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Abivax reports promising results in rheumatoid arthritis

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Abivax reports promising results in rheumatoid arthritis

March 10, 2022: “Abivax SA, a clinical-stage biotechnology company developing novel therapies that modulate the immune system to treat chronic inflammatory diseases, viral infections, and cancer, reports promising results from its phase 2a maintenance trial in rheumatoid arthritis (RA) after one year of continued daily treatment with 50mg ABX464.

Prof. Paul Emery, M.D., FMedSci, Versus Arthritis Professor of Rheumatology, Director of the Leeds Musculoskeletal Biomedical Research Centre, Leeds Teaching Hospitals Trust, Leeds Institute of Rheumatic and Musculoskeletal Medicine, UK, commented: 

“The high levels of maintained response rates within this phase 2a maintenance trial with ABX464 in rheumatoid arthritis patients, especially when it comes to ACR50 and ACR70 responses, look very promising.

The molecule also demonstrated a good safety profile, and no serious infections were observed.

Along with its very different mode of action and clinical profile, ABX464 has the potential to play an important role in the future management of rheumatoid arthritis patients.”

Prof. William Robinson, M.D., Ph.D., Chief of Division of Immunology and Rheumatology, Stanford University, US, added: “Patients suffering from chronic inflammatory diseases, such as RA, often struggle to find a suitable treatment that remains efficacious over time.

These maintenance data are very encouraging and demonstrate a potential long-term efficacy and tolerability of ABX464 for the treatment of RA, even in patients who previously did not respond or stopped responding to available therapies.”

Prof. Hartmut J. Ehrlich, M.D., CEO of Abivax, said: “These results clearly support the continued clinical development of ABX464 for the treatment of RA.

Furthermore, they are consistent with the data generated in our phase 2a and 2b ulcerative colitis trials and suggest that ABX464 has the capacity to address a broad range of chronic inflammatory indications, a disease field with a persistent high medical need and with millions of patients waiting for new, safe drugs with durable efficacy.”

After the 12-week randomized, placebo-controlled ABX464 phase 2a induction study in 60 RA patients, 67% of the patients (40/60) enrolled in the open-label extension maintenance study to receive 50mg ABX464 orally once a day for an additional 52 weeks.

58% of the patients (23/40) suffering from moderate to severe active RA completed 52 weeks of chronic treatment with ABX464.

Efficacy of 50mg once daily ABX464 was assessed by the DAS28-CRP remission (DAS28-CRP < 2.62) and the ACR20/50/70 rates:

At week 52*Full analysis set
(n=40)
(non-responder imputation)		Observed cases
(n=23)
Remission
As per DAS28-CRP < 2.6		13 (33%)13 (57%)
Low Disease Activity
As per DAS28-CRP < 3.2		17 (43%)17 (74%)
ACR2023 (58%)23 (100%)
ACR5019 (48%)19 (83%)
ACR7012 (30%)12 (52%)

            * Results based on a soft lock database review

57% of the patients (13/23) were in remission at week 52, assessed by the DAS28-CRP (< 2.6), corresponding to 33% (13/40) using the full analysis set (FAS).

All 23 patients (100%) who completed 52 weeks of treatment achieved at least an ACR20 response, which translates into 58% (23/40) in the FAS.

It is remarkable, that according to the observed cases population, 83% (19/23) and 52% (12/23) achieved even an ACR50 and ACR70 response respectively, corresponding to 48% (19/40) and 30% (12/40) according to the FAS.

17 patients discontinued the study during the first year of maintenance treatment due to either mild to moderate adverse events or worsening RA.

ABX464 was safe and the nature of the adverse events is consistent with what has been observed in more than 1,000 subjects who have so far been treated in other clinical trials with ABX464 across different indications.

At present, some UC patients have been continuously treated for four years.

ABX464 phase 2a induction and maintenance studies in rheumatoid arthritis

The placebo-controlled clinical phase 2a study was designed to evaluate the safety, tolerability and preliminary efficacy of two oral dose-levels of ABX464 administered once daily (50mg or 100mg), in combination with methotrexate (MTX).

60 patients who had an inadequate response to MTX and/or to one or more anti-tumor necrosis factor alpha (TNFα) biological therapeutics participated in this randomized and double-blind trial.

The study was conducted in 21 study centers across four European countries (France, Belgium, Poland and Hungary).

After the end of the 12-week induction study, 40 patients continued their treatment in the maintenance study with a once-daily oral dosing of 50mg ABX464.

In June 2021, Abivax communicated the results of the induction phase of its phase 2a clinical study of ABX464 administered in combination with methotrexate (MTX) for the treatment of active moderate to severe RA.

The primary endpoint of this study, safety and tolerability, was met with 50mg ABX464 once daily, demonstrating a good safety and tolerability profile in the overall patient population during the 12-week induction phase.

Epidemiology and market size in rheumatoid arthritis
In 2021, there were an estimated 3.8M diagnosed cases of rheumatoid arthritis in G7 countries (US, France, Germany, Italy, Spain, UK and Japan).

The total market size in RA is currently USD 22.3B annually, based on 2021 pharmaceutical sales estimates for rheumatoid arthritis in these countries.”

https://www.abivax.com/press-releases/

Sanofi provides update on Phase 2 study evaluating amcenestrant in breast cancer

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Sanofi provides update on Phase 2 study evaluating amcenestrant in breast cancer

March 14, 2022: “The Phase 2 AMEERA-3 clinical trial evaluating amcenestrant, an investigational optimized oral selective estrogen receptor degrader (SERD), did not meet its primary endpoint of improving progression-free survival (PFS) as assessed by an independent central review.

The trial evaluated amcenestrant as monotherapy compared to endocrine treatment of physician’s choice in patients with locally advanced or metastatic estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-) breast cancer who progressed on or after hormonal therapies.

No new safety signals were identified and the safety profile of amcenestrant in AMEERA-3 was consistent with earlier studies.

John Reed, MD, PhD
Head of Research and Development at Sanofi
“This Phase 2 trial evaluated amcenestrant as a monotherapy in a patient population with advanced disease where limited treatment options remain. 

While we are disappointed with the AMEERA-3 results, we continue to investigate amcenestrant in patients with earlier stages of breast cancer with different tumor profiles and where different standard of care treatments are used.”

Sanofi will continue to assess data from the AMEERA-3 trial and work with investigators on the publication of the full results. The ongoing clinical trial program for amcenestrant continues as planned, including AMEERA-5 and AMEERA-6.

Amcenestrant is an optimized oral SERD that binds to the estrogen receptors (ER) in breast cancer cells to inhibit their normal function and trigger degradation so they can no longer be used by tumor cells to grow.

Amcenestrant is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

About the AMEERA-3 trial

AMEERA-3 was an open-label, Phase 2 randomized trial evaluating the efficacy and safety of amcenestrant as a monotherapy compared to single-agent endocrine treatment of the physician’s choice in patients with ER+, HER2- locally advanced or metastatic breast cancer with prior exposure to hormonal therapies.

The primary objective of AMEERA-3 was to determine whether amcenestrant improved PFS assessed by an independent central review compared to endocrine monotherapy.

The key secondary efficacy endpoint was overall survival and other secondary endpoints were objective response rate, disease control rate, clinical benefit rate and duration of response.

The study also compared the overall safety profile in the two treatment arms and evaluated health-related quality of life in the two treatment arms based on patient-reported outcomes.

About the amcenestrant clinical program

The comprehensive development program for amcenestrant has been designed to evaluate its potential as an oral endocrine backbone therapy across treatment lines, including: as a single agent in second-line or later lines of treatment of ER+/HER2- metastatic breast cancer (MBC) (AMEERA-3), in combination with palbociclib in the first-line treatment of ER+/HER2- MBC (AMEERA-5), and to explore its potential in early-stage breast cancer patients in the adjuvant setting (AMEERA-6).

Initiated in late 2020, the Phase 3 AMEERA-5 clinical trial is now fully enrolled.

The Phase 3 AMEERA-6 trial, in partnership with the Breast International Group (BIG), the European Organization for Research and Treatment of Cancer (EORTC), and the Alliance Foundation Trials (AFT) is now enrolling.”

Update on US regulatory review of Fasenra in chronic rhinosinusitis with nasal polyps

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Update on US regulatory review of Fasenra in chronic rhinosinusitis with nasal polyps

 March 14, 2022: “The US FDA has issued a complete response letter (CRL) regarding the sBLA for Fasenra (benralizumab) for patients with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP).

The sBLA submitted to the FDA by AstraZeneca included data from the OSTRO Phase III trial, which met both co-primary endpoints with a safety profile consistent with the known profile of the medicine.

The CRL requested additional clinical data and the Company is working closely with the FDA regarding next steps.

The Company remains committed to bringing Fasenra to patients with CRSwNP and a second Phase III trial, ORCHID, in this indication is ongoing.

Fasenra is currently approved as an add-on maintenance treatment for severe eosinophilic asthma in the US, EU, Japan and other countries and is approved for self-administration in the US, EU and other countries.

The FDA granted Orphan Drug Designation (ODD) for Fasenra for eosinophilic granulomatosis with polyangiitis in 2018, and hypereosinophilic syndrome and eosinophilic esophagitis (EoE) in 2019.

In November 2021, the FDA also granted ODD for Fasenra for eosinophilic gastroenteritis (EGE) and eosinophilic gastritis (EG), and a Fast Track Designation for the treatment of EG with or without EGE in the US.”

FDA Orders Philips Respironics to Notify Patients Regarding Recall of Certain Breathing Assistance Machines

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FDA Orders Philips Respironics to Notify Patients Regarding Recall of Certain Breathing Assistance Machines

March 10, 2022: “The U.S. FDA issued a notification order to Philips Respironics requiring the company to notify patients and others of the company’s June 14, 2021, recall of certain Philips Respironics ventilators, continuous positive airway pressure (CPAP) and bilevel positive airway pressure (BiPAP) machines, and the unreasonable risk of substantial harm to the public health posed by the degradation of the polyester-based polyurethane (PE-PUR) sound abatement foam used in those products.

The FDA has determined that this order is necessary to eliminate the unreasonable risk of harm posed by the recalled products, because the company’s notification efforts to date have been inadequate

The FDA has heard the frustration expressed by patients and durable medical equipment suppliers who are unaware of the recall and have received insufficient information on their next steps regarding the recall process,” said Jeff Shuren, M.D., J.D., director of the FDA’s Center for Devices and Radiological Health.

“Taking this action today enables the FDA to mandate that Philips Respironics improve its communication about the recall and the serious risk posed by the foam used in the recalled products with patients and the public and to ensure that individuals who rely on these essential devices are receiving the important information they need from the company.”  

The FDA is ordering Philips Respironics to notify all device users, durable medical equipment (DME) suppliers, distributors, retailers, and health care providers who prescribe the products about the recall and the health risks posed by the foam used in the recalled products.

The order also directs Philips to maintain language to patients regarding the risk of using ozone cleaners on the recalled devices on their main webpage for the recall, and to provide instructions for device users to register their devices on the Philips website. 

Along with these actions, the FDA recommends additional measures Philips can take to better communicate with the public regarding the recall.

Specifically, the FDA recommends that Philips provide monthly updates to device users who register their devices on the Philips website that include information on expected time for replacement and current rate of replacement of recalled devices.

The FDA also recommends that Philips provide detailed information to device users, DME suppliers, distributors, retailers, and healthcare providers on the replacement process. 

In June 2021, Philips Respironics initiated a recall of certain ventilators, CPAP and BiPAP machines due to potential health risks related to PE-PUR sound abatement foam used in those devices.

This particular foam may break down and can result in serious injury, which can be life-threatening, cause permanent impairment and/or require medical intervention to prevent permanent injury to users. 

Since the initiation of the recall, the FDA has engaged with Philips on several fronts about the effectiveness of its communications with the public regarding the recall and the risks presented by the recalled products and has expressed concern that it is likely a significant portion of patients and consumers using the recalled products are unaware of the health risks presented by those products. “

The FDA is issuing this order under section 518(a) of the Federal Food, Drug, and Cosmetic Act. Under this authority, the FDA may order a manufacturer to provide notification if the FDA determines that a device presents an “unreasonable risk of substantial harm to the public health” and such notification is necessary to “eliminate the unreasonable risk of such harm and no more practical means are available [under the Federal Food, Drug, and Cosmetic Act] to eliminate such risk.”

The FDA may order that adequate notification be provided to device users, health care providers who prescribe or use the device, and any other person who should receive notification to eliminate such risk. 

The order directs Philips to provide the notification described in the order within 45 days of its issuance.”

https://www.fda.gov/news-events/press-announcements/fda-orders-philips-respironics-notify-patients-regarding-recall-certain-breathing-assistance

Pfizer Initiates Phase 2/3 Study of Novel COVID-19 Oral Treatment in Pediatric Participants

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Pfizer Initiates Phase 2/3 Study of Novel COVID-19 Oral Treatment in Pediatric Participants

March 09, 2022: “Pfizer Inc. announced that it has initiated a Phase 2/3 study, EPIC-PEDS (Evaluation of Protease Inhibition for COVID-19 in Pediatric Patients), to evaluate the safety, pharmacokinetics, and efficacy of Pfizer’s PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) in non-hospitalized, symptomatic, pediatric participants with a confirmed diagnosis of COVID-19 who are at risk of progression to severe disease.

“Since the beginning of the pandemic, more than 11 million children under the age of 18 in the United States alone have tested positive for COVID-19, representing nearly 18% of reported cases and leading to more than 100,000 hospital admissions.

There is a significant unmet need for outpatient treatments that can be taken by children and adolescents to help prevent progression to severe illness, including hospitalization or death,” said Mikael Dolsten, Chief Scientific Officer and President, Worldwide Research, Development and Medical, Pfizer.

“PAXLOVID is already authorized or approved in many countries around the world, with more than 1.5 million treatment courses delivered thus far and 30 million expected by July to help combat this devastating disease.

We are proud to expand studies of our novel COVID-19 treatment to include pediatric participants to further evaluate the safety and efficacy of this treatment in this important population.”

The Phase 2/3 trial is an open-label, multi-center, single-arm study in approximately 140 pediatric participants under 18 years of age.

Initial enrollment features two cohorts; Cohort 1 includes participants aged 6 to 17 weighing at least 40 kg [88 lbs], and Cohort 2 includes those aged 6 to 17 weighing more than 20 kg [44 lbs] and less than 40 kg [88 lbs].

  • Participants enrolled in Cohort 1 will receive PAXLOVID (nirmatrelvir/ritonavir 300 mg/100 mg) orally twice daily for five days (10 doses total), the current authorized dosing for pediatric patients 12 years of age and older weighing at least 40kg.
  • Participants enrolled in Cohort 2 will receive PAXLOVID (nirmatrelvir/ritonavir 150 mg/100 mg) orally twice daily for five days (10 doses total).

Pfizer is also working to develop an age-appropriate formulation for three additional planned cohorts of younger than 6 years old and will enroll the trial to include these younger age groups as data from Cohorts 1 and 2 and the new formulation are available.

An independent Data Monitoring Committee (DMC) will review safety data of participants in each cohort.

Data from the Phase 2/3 study of non-hospitalized, high-risk adults with COVID-19 showed PAXLOVID reduced risk of hospitalization or death by 89% (within three days of symptom onset) and 88% (within five days of symptom onset) from any cause compared to placebo, with no deaths observed in the treatment group.

Treatment-emergent adverse events were comparable between PAXLOVID (23%) and placebo (24%), most of which were mild in intensity.

The safety and effectiveness of PAXLOVID have not yet been directly established in pediatric patients.

Although other PAXLOVID clinical trials did not include participants under the age of 18, the FDA authorized PAXLOVID for emergency use in pediatric patients 12 years of age and older weighing at least 40 kg [88lbs] as pharmacokinetic-pharmacodynamic (PK/PD) modeling determined that the authorized adult dosing regimen would result in comparable blood concentration levels of PAXLOVID in this population and the adults with similar body weight who were included in the EPIC-HR trial.

Data from the EPIC-PEDS study will provide further support for the dose recommendations in this population, as well as potentially expand the indication to younger age groups and lower weights.

PAXLOVID is currently authorized or approved in more than 50 countries across the globe.

Please see Full Emergency Use Authorization (EUA) Prescribing Information available at www.fda.gov and www.COVID19oralRx.com.

Our Commitment to Equitable Access

Pfizer is committed to working toward equitable access to PAXLOVID for all people, aiming to deliver safe and effective antiviral therapeutics as soon as possible and at an affordable price.

During the pandemic, Pfizer will offer its oral therapy through a tiered pricing approach, pending country authorization or approval, based on the income level of each country to promote equity of access across the globe.

High and upper-middle income countries will pay more than lower income countries.

Pfizer continues to invest to support the manufacturing and distribution of PAXLOVID, including exploring potential contract manufacturing options.

As a result of these efforts, Pfizer has raised its production projections, with the ability to produce up to 120 million courses of treatment by the end of 2022, pending global demand.

The company has initiated bilateral outreach to more than 100 countries around the world and has entered into agreements with multiple countries.

Additionally, Pfizer has signed a voluntary license agreement with the Medicines Patent Pool (MPP) for its oral treatment to help expand access, pending country regulatory authorization or approval, in 95 low- and middle-income countries that account for approximately 53% of the world’s population.

About PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets)

PAXLOVID is a SARS-CoV-2 main protease (Mpro) inhibitor (also known as SARS-CoV-2 3CL protease inhibitor) therapy.

It was developed to be administered orally so that it can be prescribed at the first sign of infection or, pending clinical success of the rest of the EPIC development program and subject to regulatory authorization, at first awareness of an exposure – potentially helping patients avoid severe illness (which can lead to hospitalization and death) or avoid disease development following contact with a household member who contracts COVID-19.

Nirmatrelvir [PF-07321332], which originated in Pfizer laboratories, is designed to block the activity of the Mpro, an enzyme that the coronavirus needs to replicate.

Co-administration with a low dose of ritonavir helps slow the metabolism, or breakdown, of nirmatrelvir in order for it to remain active in the body for longer periods of time at higher concentrations to help combat the virus.

Nirmatrelvir is designed to inhibit viral replication at a stage known as proteolysis, which occurs before viral RNA replication.

In preclinical studies, nirmatrelvir did not demonstrate evidence of mutagenic DNA interactions.

Current variants of concern can be resistant to treatments that work by binding to the spike protein found on the surface of the SARS-CoV-2 virus.

PAXLOVID, however, works intracellularly by binding to the highly conserved Mpro of the SARS-CoV-2 virus to inhibit viral replication.

Nirmatrelvir has shown consistent in vitro antiviral activity against earlier and current variants of concern (i.e., Alpha, Beta, Delta, Gamma, Lambda, Mu, and Omicron).

PAXLOVID is generally administered at a dose of 300 mg (two 150 mg tablets) of nirmatrelvir with one 100 mg tablet of ritonavir, given twice-daily for five days.

One carton contains five blister packs of PAXLOVID, as co-packaged nirmatrelvir tablets with ritonavir tablets, providing all required doses for a full five-day treatment course.

About the EPIC Development Program

The EPIC (Evaluation of Protease Inhibition for COVID-19) Phase 2/3 development program for PAXLOVID consists of four clinical trials spanning a broad spectrum of participants, including adults who have been exposed to the virus through household contacts, adults at both standard risk and high risk of progressing to severe illness, and children under the age of 18 at risk of progressing to severe disease.​

In July 2021, Pfizer initiated the first of these trials, known as EPIC-HR (Evaluation of Protease Inhibition for COVID-19 in High-Risk Patients), a randomized, double-blind study of non-hospitalized adults with COVID-19, who are at high risk of progressing to severe illness.

At the recommendation of an independent Data Monitoring Committee and in consultation with the U.S. FDA, Pfizer ceased further enrollment into the study in early November 2021 due to the overwhelming efficacy demonstrated in these results.

Findings from the EPIC-HR final analysis were published online in The New England Journal of Medicineon February 16, 2022.

In August 2021, Pfizer began the Phase 2/3 EPIC-SR (Evaluation of Protease Inhibition for COVID-19 in Standard-Risk Patients) study to evaluate efficacy and safety in adults with a confirmed diagnosis of SARS-CoV-2 infection who are at standard risk (i.e., low risk of hospitalization or death).

Interim data from this study have been reported. Pfizer is currently expanding the population of the ongoing EPIC-SR study by approximately 800 participants and expects to share results later this year.

In September 2021, Pfizer initiated the Phase 2/3 EPIC-PEP (Evaluation of Protease Inhibition for COVID-19 in Post-Exposure Prophylaxis) study to evaluate efficacy and safety in adults exposed to SARS-CoV-2 by a household member. This trial is also ongoing, and Pfizer expects to share results later this year.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-initiates-phase-23-study-novel-covid-19-oral

Bayer submits applications in the U.S. and EU for additional indication of Nubeqa™

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Bayer submits applications in the U.S. and EU for additional indication of Nubeqa™

 March 9, 2022: “Bayer announced the submission of a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) as well as the submission of a Variation Type II application to the European Medicines Agency (EMA) for the oral androgen receptor inhibitor (ARi) darolutamide.

Bayer is seeking approval for the use of darolutamide in combination with docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC).

The compound is already approved in more than 60 markets around the world, including the U.S., the European Union (EU), Japan and China, under the brand name Nubeqa™, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease.

The submissions are supported by positive results from the Phase III ARASENS trial, showing a statistically significant improvement in overall survival (OS) for darolutamide plus androgen deprivation therapy (ADT) and docetaxel in men with mHSPC.

These results were presented in February at the 2022 ASCO GU Cancers Symposium and simultaneously published in The New England Journal of Medicine.

“For men with mHSPC, there remains a high need for new treatment options that can extend overall survival and delay the progression to CRPC,” said Christine Roth, Member of the Executive Committee of Bayer’s Pharmaceutical Division and Head of the Oncology SBU at Bayer.

“Prostate cancer is a key area of focus at Bayer and the U.S. and EU submissions for darolutamide in mHSPC represent a significant milestone in our commitment to developing treatments that support men with prostate cancer throughout the different stages of the disease.”

Darolutamide is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company. Additional submissions in mHSPC are planned globally.

The compound is also being investigated in further studies across various stages of prostate cancer, including another Phase III trial in mHSPC (ARANOTE) as well as an ANZUP-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801).

About the ARASENS Trial
The ARASENS trial is the only randomized, Phase III, multi-center, double-blind, trial which was prospectively designed to compare the use of a second-generation oral androgen receptor inhibitor (ARi) plus androgen deprivation therapy (ADT) and docetaxel to ADT plus docetaxel (a guideline recommended standard-of-care) in metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo, plus ADT and docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all measured at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability.

About Metastatic Hormone-Sensitive Prostate Cancer
Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease.

Approximately 5% of men will already suffer from prostate cancer with distant metastases when first diagnosed.

Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of the chemotherapy docetaxel and ADT.

Despite these treatments, a large proportion of men with mHSPC will eventually progress to metastatic castration-resistant prostate cancer (mCRPC), a condition with high morbidity and limited survival.

About Nubeqa™ (darolutamide)
Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells.

The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans.

A low blood-brain barrier penetration would explain the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS Phase III trial and the improved verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.

The product is approved under the brand name Nubeqa™ in more than 60 markets around the world, including the U.S., EU, Japan, China, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease.

The compound is also being investigated in further studies across various stages of prostate cancer, including another Phase III trial in mHSPC (ARANOTE) as well as the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP)-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov.

About Prostate Cancer at Bayer
Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments.

The company has the passion and determination to develop new medicines that help improve and extend the lives of people living with cancer. Prostate cancer is the second most commonly diagnosed cancer in men1 and a key area of focus for Bayer.

The company’s franchise includes two products on the market (Nubeqa™ and Xofigo™) and several compounds in development, including a unique approach of advancing targeted alpha therapies.

Bayer is focused on addressing the unique needs of prostate cancer patients, providing treatments that extend their lives throughout the different stages of the disease and allowing them to continue their everyday activities, so that they can live longer, better lives.”

https://media.bayer.com/baynews/baynews.nsf/id/Bayer-submits-applications-in-the-US-and-EU-for-additional-indication-of-Nubeqa-darolutamide?OpenDocument&sessionID=1646816763

Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio

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Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio

March 8, 2022: “Roche announced that new data from its growing neuromuscular portfolio will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, 13 – 16 March 2022.

Presentations include eight abstracts from its spinal muscular atrophy (SMA) programme and three from its Duchenne muscular dystrophy (DMD) programme, demonstrating Roche’s commitment to advancing clinical understanding of these conditions with the aim of treating a broad range of people living with neuromuscular disorders.

“These new data further highlight the compelling body of evidence for Evrysdi across infants, children and adults living with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development.

“The results from our Duchenne Muscular Dystrophy gene therapy programme are encouraging and represent the potential to provide transformative outcomes for people living with this condition. We are grateful for the partnerships that can help us accelerate progress across SMA and DMD.”

Spinal Muscular Atrophy (SMA)
Six abstracts from the Evrysdi® (risdiplam) clinical development programme will be presented.

This includes 3-year SUNFISH Part 1 and 2 efficacy and safety data, as well as a comparison of 2-year SUNFISH Part 2 data with an untreated external control group, both of which highlight the long-term efficacy and safety profile of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA.

The data also demonstrate sustained increase in motor function over time, compared to an untreated external control group at 2-years.

Updated interim data from the RAINBOWFISH study will report updated safety data in enrolled infants and efficacy data in infants who have received risdiplam for at least 12 months.

Roche will also be presenting data from four other SMA studies including:

  • 24-month data from FIREFISH Part 1 and 2 in SMA Type 1, exploring the safety and efficacy of Evrysdi in infants treated with Evrysdi for 2-years
  • 12-month data from the JEWELFISH study, exploring the safety, tolerability and pharmacokinetic/pharmacodynamic (PD) relationship of Evrysdi in a broad age range of patients (6 months to 60 years) who have previously received RG7800 (RO6885247), nursinersen, olesoxime or onasemnogene abeparvovec
  • Design of new MANATEE trial, a multi-centre, randomised, placebo-controlled, double-blind study investigating the effect of GYM329, an investigational anti-myostatin, in combination with Evrysdi
  • Data on the demographics and clinical characteristics of people with SMA enrolled in the Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) hub
  • Using baseline data from FIREFISH, SUNFISH, JEWELFISH and NAtHis-SMA studies, a validated algorithm for estimating the weight of patients with SMA based on few input parameters

Duchenne Muscular Dystrophy (DMD)
Roche has partnered with Sarepta to accelerate access to investigational gene therapy, delandistrogene moxeparvovec (SRP-9001), for DMD outside of the United States upon US Food and Drug Adminstration (FDA) approval.

Sarepta, will present three year data from Study 101, an open-label Phase 1/2a study evaluating the safety of SRP-9001 in four ambulatory participants aged between 4-7 years old with DMD.

The results show an acceptable safety profile, and all patients demonstrated a clinically meaningful improvement in their North Star Ambulatory Assessment (NSAA) from baseline.

In addition, Sarepta will also present results from Study 102 which continue to support SRP-9001’s clinical profile and reinforce a potential benefit risk profile. Study 102 is a three-part, Phase 2 clinical study evaluating the safety and efficacy of delandistrogene moxeparvovec in patients with DMD. Analysis of Part 2 of the study will be presented.

Other presentations include:

The full range of data from Roche’s clinical development programme in neuroscience being presented at MDA 2022 include:

Medicine Abstract TitlePresentation Number (type), Presentation Date, Time
Evrysdi® (risdiplam) for spinal muscular atrophy 

 

 

 

 

 

 

 

 

 		SUNFISH Parts 1 and 2: 3-year efficacy and safety of risdiplam in Types 2 and 3 SMAData presentation 
Wednesday 16th March 
8:20 – 8:35 am CT (14:20 – 14:35 CET)
SUNFISH Part 2: 24-month efficacy of risdiplam compared with external control comparatorsPosters 
Sunday 13th March 
18:00 – 20:00 CT
(02:00 – 04:00 am CET) 

 

Monday 14th March 
10:00 am – 20:00 CT (16:00 – 02:00 am CET)
Tuesday 15th March 
10:00 am – 20:00 CT (16:00 – 02:00 am CET)

 

 

Dedicated Poster sessions between 6:00-8:00pm daily




 

 

 

 

 

 

 

 

 

 

 
MANATEE: A study of GYM329 (RO7204239) in combination with risdiplam treatment in pediatric patients with SMA
Demographics and clinical characteristics of individuals with spinal muscular atrophy enrolled in the Muscular Dystrophy Association MOVR data hub
FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA
JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving treatment with risdiplam
RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA
Statistical modelling to estimate patients’ weight in Types 1−3 SMA
SRP-9001 for Duchenne muscular dystrophy 

 

 		EMBARK study design: Phase 3 trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy
Phase 1/2a trial of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy: 3-year safety and functional outcomes
A Phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy

Full session details and data presentations listing for the MDA Clinical and Scientific Conference can be found at the meeting website: https://mdaconference.org/

Follow Roche on Twitter via @Roche and keep up to date with MDA 2022 Conference news and updates by using the hashtag #RocheAtMDA2022, #MDA and #neuromuscular

About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.

To date over 5,000 patients have been treated with Evrysdi, which is administered daily at home in liquid form by mouth or by feeding tube.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues.

SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S Food and Drug Administration in 2017.

In 2021 Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery.

Evrysdi is currently approved in 75 countries and the dossier is under review in a further 27 countries.”

https://www.roche.com/media/releases/med-cor-2022-03-08

Voyager Therapeutics and Novartis supports therapies for previously untreatable brain regions

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Voyager Therapeutics and Novartis supports therapies for previously untreatable brain regions

March 08, 2022: “Voyager Therapeutics, a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids announced an agreement through which Novartis may exercise options to license novel AAV capsids generated from Voyager’s RNA-driven TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform for potential use with three undisclosed CNS targets and options to access capsids for two additional targets to be agreed on in the future.

Voyager will receive $54 million upfront and is entitled to receive up to $37.5 million in exercise fees for options for three initial CNS targets, exercisable by Novartis within 12 months of signing.

In addition, Novartis may elect to evaluate capsids for up to two additional targets to be agreed on in the future, subject to their availability, for $18 million upon selection of each target, and a $12.5 million exercise fee for selection of a capsid for each target.

Voyager is also eligible to earn up to $1.5 billion in potential development, regulatory, and commercial milestones for products utilizing Voyager licensed capsids, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.

“This transaction further validates the potential of the Voyager TRACER capsid discovery platform to broadly enable a next generation of AAV gene therapies,” said Allen Nunnally, chief business officer of Voyager.

“Our collaboration with Novartis expands the array of therapeutic programs in which our proprietary capsids may be deployed and highlights the potential of our TRACER platform to generate future business development opportunities as our novel capsid library expands and initial TRACER-derived capsids are further refined to enhance desirable characteristics.”

“Overcoming the substantial toxicity risk frequently observed with high doses of AAV-based gene therapies remains a critical challenge to realizing the full, curative potential of this breakthrough modality,” said Glenn Pierce, M.D., Ph.D., interim chief scientific officer of Voyager.

“Voyager’s growing and maturing library of proprietary TRACER-derived capsids have demonstrated markedly enhanced expression in non-human primates with more precise targeting of desired tissue and cell types, creating the potential for superior delivery and fewer off-target risks than conventional AAV.”

Under the terms of the agreement, Novartis has the right to evaluate novel capsids from Voyager’s TRACER platform and to exercise options to license capsids for exclusive use with specific targets in Novartis’ development of AAV gene therapies.

The targets for which Novartis receives rights under the agreement are distinct from those in Voyager’s internal and partnered pipeline, and Voyager retains global rights to its TRACER discovery platform as well as all capsids arising from it for use with other targets, subject to Voyager’s obligations under its agreement with Pfizer announced in October 2021 for access to TRACER capsids for use with one neurologic and one cardiovascular target.

An initial set of proprietary AAV capsids derived from Voyager’s TRACER platform have demonstrated superior blood-brain barrier penetration, increased transduction in the brain and spinal cord, enhanced cardiac muscle tropism, and increased transgene expression in target tissues compared to conventional AAV capsids when dosed intravenously in non-human primates (NHPs).

  • A capsid from this initial set demonstrated more than 1,000-fold increased transgene expression levels (as measured by mRNA level) compared to conventional AAV9 across a wide array of brain regions when dosed intravenously in NHPs.
  • A separate capsid showed significantly enhanced cardiac muscle transduction and dorsal root ganglia detargeting compared to conventional AAV9 when dosed intravenously in NHPs.

Subsequent TRACER screening campaigns have identified a new class of AAV9 variants selective for glial cells, which may enable more precise targeting of CNS diseases affecting non-neuronal cells, as well as AAV5 capsid variants with enhanced brain and spinal cord tropism, compared to conventional AAV9 and AAV5.

Voyager is performing further screening with its TRACER capsid discovery platform to identify additional proprietary AAV9- and AAV5-derived capsids targeting multiple tissue and cell types, as well as further refining identified capsids to enhance desirable characteristics for use in gene therapies to treat a broad range of diseases.

Conference Call
The Voyager Therapeutics leadership team will host a conference call and webcast today at 8:30 a.m.

ET to discuss the Novartis license option agreement, and provide and discuss fourth quarter and full year 2021 financial and operating results. To access the call, please dial (877) 851-3834 (domestic) or (631) 291-4595 (international) and ask for the Voyager Therapeutics earnings call.

A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay will be available at the same link approximately two hours after its completion.

The replay will be available for at least 30 days following the conclusion of the call.

About the TRACERTM AAV Capsid Discovery Platform
Voyager’s TRACERTM capsid discovery platform is a broadly applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV9- and AAV5-derived capsids with enhanced tropisms and cell- and tissue-specific transduction properties in multiple species, including non-human primates (NHPs).

Initial data from the first of many libraries screened in NHPs demonstrated the proprietary capsid variants effectively penetrated the blood-brain barrier and achieved widespread biodistribution and transduction of multiple regions of the brain.

Separate results have demonstrated the ability of certain capsids to transduce cardiac muscle and to de-target the dorsal root ganglia.

Voyager is proceeding with additional capsid campaigns derived from AAV9, AAV5, and other capsid serotypes to identify novel AAV vectors optimized for specific therapeutic applications.”

https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-announces-license-option-agreement-novartis

Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio

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Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio

March 8, 2022: “Roche announced that new data from its growing neuromuscular portfolio will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, 13 – 16 March 2022.

Presentations include eight abstracts from its spinal muscular atrophy (SMA) programme and three from its Duchenne muscular dystrophy (DMD) programme, demonstrating Roche’s commitment to advancing clinical understanding of these conditions with the aim of treating a broad range of people living with neuromuscular disorders.

“These new data further highlight the compelling body of evidence for Evrysdi across infants, children and adults living with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development.

“The results from our Duchenne Muscular Dystrophy gene therapy programme are encouraging and represent the potential to provide transformative outcomes for people living with this condition.

We are grateful for the partnerships that can help us accelerate progress across SMA and DMD.”

Spinal Muscular Atrophy (SMA)
Six abstracts from the Evrysdi® (risdiplam) clinical development programme will be presented.

This includes 3-year SUNFISH Part 1 and 2 efficacy and safety data, as well as a comparison of 2-year SUNFISH Part 2 data with an untreated external control group, both of which highlight the long-term efficacy and safety profile of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA.

The data also demonstrate sustained increase in motor function over time, compared to an untreated external control group at 2-years.

Updated interim data from the RAINBOWFISH study will report updated safety data in enrolled infants and efficacy data in infants who have received risdiplam for at least 12 months.

Roche will also be presenting data from four other SMA studies including:

  • 24-month data from FIREFISH Part 1 and 2 in SMA Type 1, exploring the safety and efficacy of Evrysdi in infants treated with Evrysdi for 2-years
  • 12-month data from the JEWELFISH study, exploring the safety, tolerability and pharmacokinetic/pharmacodynamic (PD) relationship of Evrysdi in a broad age range of patients (6 months to 60 years) who have previously received RG7800 (RO6885247), nursinersen, olesoxime or onasemnogene abeparvovec
  • Design of new MANATEE trial, a multi-centre, randomised, placebo-controlled, double-blind study investigating the effect of GYM329, an investigational anti-myostatin, in combination with Evrysdi
  • Data on the demographics and clinical characteristics of people with SMA enrolled in the Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) hub
  • Using baseline data from FIREFISH, SUNFISH, JEWELFISH and NAtHis-SMA studies, a validated algorithm for estimating the weight of patients with SMA based on few input parameters

Duchenne Muscular Dystrophy (DMD)
Roche has partnered with Sarepta to accelerate access to investigational gene therapy, delandistrogene moxeparvovec (SRP-9001), for DMD outside of the United States upon US Food and Drug Adminstration (FDA) approval.

Sarepta, will present three year data from Study 101, an open-label Phase 1/2a study evaluating the safety of SRP-9001 in four ambulatory participants aged between 4-7 years old with DMD.

The results show an acceptable safety profile, and all patients demonstrated a clinically meaningful improvement in their North Star Ambulatory Assessment (NSAA) from baseline.

In addition, Sarepta will also present results from Study 102 which continue to support SRP-9001’s clinical profile and reinforce a potential benefit risk profile.

Study 102 is a three-part, Phase 2 clinical study evaluating the safety and efficacy of delandistrogene moxeparvovec in patients with DMD.

Analysis of Part 2 of the study will be presented.

Other presentations include:

  • The study design and methodology of the first delandistrogene moxeparvovec global Phase III gene therapy trial, EMBARK, assessing the safety and efficacy of commercially representative delandistrogene moxeparvovec material in ambulatory boys with a confirmed DMD mutation aged 4 to 7 years old.

The full range of data from Roche’s clinical development programme in neuroscience being presented at MDA 2022 include:

Medicine Abstract TitlePresentation Number (type), Presentation Date, Time
Evrysdi® (risdiplam) for spinal muscular atrophy 

 

 

 

 

 

 

 

 

 		SUNFISH Parts 1 and 2: 3-year efficacy and safety of risdiplam in Types 2 and 3 SMAData presentation 
Wednesday 16th March 
8:20 – 8:35 am CT (14:20 – 14:35 CET)
SUNFISH Part 2: 24-month efficacy of risdiplam compared with external control comparatorsPosters 
Sunday 13th March 
18:00 – 20:00 CT
(02:00 – 04:00 am CET) 

 

Monday 14th March 
10:00 am – 20:00 CT (16:00 – 02:00 am CET)
Tuesday 15th March 
10:00 am – 20:00 CT (16:00 – 02:00 am CET)

 

 

Dedicated Poster sessions between 6:00-8:00pm daily




 

 

 

 

 

 

 

 

 

 

 
MANATEE: A study of GYM329 (RO7204239) in combination with risdiplam treatment in pediatric patients with SMA
Demographics and clinical characteristics of individuals with spinal muscular atrophy enrolled in the Muscular Dystrophy Association MOVR data hub
FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA
JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving treatment with risdiplam
RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA
Statistical modelling to estimate patients’ weight in Types 1−3 SMA
SRP-9001 for Duchenne muscular dystrophy 

 

 		EMBARK study design: Phase 3 trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy
Phase 1/2a trial of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy: 3-year safety and functional outcomes
A Phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy

Full session details and data presentations listing for the MDA Clinical and Scientific Conference can be found at the meeting website: https://mdaconference.org/

Follow Roche on Twitter via @Roche and keep up to date with MDA 2022 Conference news and updates by using the hashtag #RocheAtMDA2022, #MDA and #neuromuscular”

https://www.roche.com/media/releases/med-cor-2022-03-08

FDA Makes Low-Risk Determination for Marketing of Products from Genome-Edited Beef Cattle After Safety Review

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FDA Makes Low-Risk Determination for Marketing of Products from Genome-Edited Beef Cattle After Safety Review

March 07, 2022: “The U.S. FDA announced it has made a low-risk determination for the marketing of products, including food, from two genome-edited beef cattle and their offspring after determining that the intentional genomic alteration (IGA) does not raise any safety concerns (low-risk determination).

The IGA results in the equivalent genotype (genetic make-up) and short-hair coat trait seen in some conventionally bred cattle, known as a “slick” coat. This is the FDA’s first low-risk determination for enforcement discretion for an IGA in an animal for food use. 

“Today’s decision underscores our commitment to using a risk and science-based, data-driven process that focuses on safety to the animals containing intentional genomic alterations and safety to the people who eat the food produced by these animals,” said Steven M. Solomon, D.V.M., M.P.H., director of the FDA’s Center for Veterinary Medicine.

“It also demonstrates our ability to identify low-risk IGAs that don’t raise concerns about safety, when used for food production.

We expect that our decision will encourage other developers to bring animal biotechnology products forward for the FDA’s risk determination in this rapidly developing field, paving the way for animals containing low-risk IGAs to more efficiently reach the marketplace.”

Based on the agency’s review of scientific data, the FDA has determined that the product is low-risk and does not raise any safety concerns, and the FDA does not expect the product developer of the IGA to pursue the FDA’s approval prior to marketing (enforcement discretion).

To date, the FDA has made low-risk determinations for enforcement discretion for many other IGAs in animals for non-food uses and also has approved applications for five IGAs: in groups of goatExternal Link Disclaimer, chickenExternal Link Disclaimer, salmonExternal Link Disclaimer, rabbit and, most recently, in a line of pigs.

IGAs are alterations made using molecular technologies that introduce changes to the genome of an animal.

The IGA in these cattle, known as PRLR-SLICK cattle, was introduced using a genome-editing technique known as CRISPR.

The IGA can be passed on to offspring, allowing the trait to be shared through conventional breeding.

There are conventionally bred cattle with naturally-occurring mutations that result in the same extremely short, slick-hair coat.

Reports in scientific literature indicate that cattle with this extremely short, slick-hair coat are potentially able to better withstand hot weather. Cattle that are comfortable in their environment are less likely to experience temperature-related stress and may result in improved food production.

Although PRLR-SLICK cattle have an equivalent trait to those cattle with a naturally-occurring short hair coat, they are not currently in commerce.

The product developer plans to use the genetic products from these two animals with select customers in the global market soon and anticipates meat products will be available for purchase by general consumers as early as two years.

The FDA reviewed genomic data and other information submitted by the product developer confirming that the IGA in genome-edited PRLR-SLICK cattle is equivalent to naturally occurring mutations that have arisen in several breeds of cattle as an adaptation to being raised in tropical or subtropical environments.

The data also confirmed that the IGA results in the same slick-hair trait as in cattle found in conventional agriculture.

Further, the food from the cattle is the same as food from conventionally bred cattle that have the same slick-hair trait.

The FDA does not expect farms or facilities not owned or operated by the developer that are producing and breeding these low-risk PRLR-SLICK cattle using conventional breeding techniques to register with the agency.

The low-risk determination was provided to Acceligen.”

https://www.fda.gov/news-events/press-announcements/fda-makes-low-risk-determination-marketing-products-genome-edited-beef-cattle-after-safety-review

FDA Urges Companies to be ’Recall Ready’ to Protect Public Health as Part of Final Guidance for Voluntary Recalls

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FDA Urges Companies to be ’Recall Ready’ to Protect Public Health as Part of Final Guidance for Voluntary Recalls

March 03, 2022: “The U.S. Food and Drug Administration finalized guidance to help companies prepare to quickly and effectively remove violative products from the market.

The guidance describes steps companies should take, before a recall is necessary, to develop recall policies and procedures that include training, planning and record-keeping to reduce the time a recalled product is on the market and, thus, limit the public’s exposure to risk. 

“Voluntary recalls continue to be the fastest, most effective way for a company to correct or remove violative and potentially harmful products from the market to help keep consumers safe,” said Associate Commissioner of Regulatory Affairs Judith McMeekin, Pharm. D.

“It is critical that all companies in the supply chain are ‘recall ready’ to ensure appropriate actions are taken swiftly across the distribution channels to best protect public health and the integrity of the supply chain.

We will continue to work with companies to improve their recall procedures and minimize Americans’ exposure to potentially harmful products.”   

A voluntary recall is an action taken by a company to correct a violative product or remove it from the market.

A recalling company may act on its own initiative or the FDA may inform the company that a distributed product violates the law and recommend the company recall the product.

The FDA has the authority to require recalls of certain products in particular circumstances, such as controlled substances, biological products, human cells, tissues and cellular and tissue-based products, medical devices and foods. 

The final guidance, Initiation of Voluntary Recalls Under 21 CFR Part 7, Subpart C, explains how companies should have adequate product coding and maintain distribution records to facilitate faster, more accurate recall actions, which the FDA recommends that all companies do.

It encourages recalling companies to use electronic communications to quickly identify and provide certain product information when alerting consignees and the public about a voluntary recall. 

The guidance also urges companies to act quickly to initiate a recall when public health is at risk and to do so prior to completing an investigation into the cause of the problem.

Because recalls can affect the entire supply chain, including downstream suppliers, wholesalers or vendors, the FDA recommends that companies develop recall procedures to quickly inform their entire distribution chain, so consignees can rapidly identify affected lots and recall downstream products when necessary. 

This latest action reflects the agency’s ongoing commitment to work closely with companies so they can quickly act to protect the public from violative products on the market.

In recent years, the FDA has made proactive and systemic improvements to recall processes, including issuing guidance on public availability of lists of retail consignees to effectuate certain human and animal food recalls; mandatory recalls for human and animal foods; and public warnings and notifications for all FDA-regulated products, which reiterates a policy to rapidly post new recalls to the FDA’s weekly Enforcement Reports, a public listing of all recalls monitored by the FDA.”

https://www.fda.gov/news-events/press-announcements/fda-urges-companies-be-recall-ready-protect-public-health-part-final-guidance-voluntary-recalls

Nirsevimab significantly protected infants against RSV disease in Phase III MELODY trial

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Nirsevimab significantly protected infants against RSV disease in Phase III MELODY trial

 March 03, 2022: “Detailed results from the positive MELODY Phase III trial showed a single dose of AstraZeneca and Sanofi’s nirsevimab met the primary efficacy endpoint reducing the incidence of medically attended lower respiratory tract infections (LRTI) caused by respiratory syncytial virus (RSV) by 74.5% (95% CI 49.6, 87.1; p<0.001), compared to placebo.

The trial involved healthy term and late preterm (gestational age ≥35 weeks) infants entering their first RSV season.

Additionally, the MEDLEY Phase II/III trial, which evaluated safety and pharmacokinetics of nirsevimab in infants with congenital heart disease (CHD), chronic lung disease (CLD) and prematurity entering their first RSV season, demonstrated nirsevimab had a similar safety and tolerability profile compared to Synagis (palivizumab).

Serum levels of nirsevimab following dosing (on day 151) in this trial were comparable with those observed in the MELODY Phase III trial, indicating similar protection in this population to that in the healthy term and late preterm infants is likely.

Synagis is currently the only available preventative option for RSV.

Nirsevimab is an investigational long-acting antibody designed to protect all infants through their first RSV season with a single dose.

It is the first potential immunisation to show protection against RSV in the general infant population in a Phase III trial.

Dr William Muller, Associate Professor, Pediatrics, Northwestern University Feinberg School of Medicine and Scientific Director, Clinical and Community Trials, Ann & Robert H. Lurie Children’s Hospital of Chicago, Illinois, US and primary investigator of the MELODY Phase III trial, said: “We know that respiratory syncytial virus has seen a resurgence with the easing of COVID-19 public health measures.

This shows us a broad immunization approach is needed to help mitigate the substantial global burden respiratory syncytial virus places on infants, their families and healthcare services.

These exciting data show that nirsevimab has the potential to offer RSV protection for all infants, which would be a paradigm shift in the approach to this disease.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Respiratory syncytial virus is a leading cause of lower respiratory tract infections, such as bronchiolitis or pneumonia, as well as hospitalisations in infants.

These data show for the first time, the potential to significantly protect all infants through their first respiratory syncytial virus season with a single dose immunisation and we look forward to working with health authorities to bring nirsevimab to infants as quickly as possible.”

Jean-François Toussaint, Global Head of Research and Development Vaccines, Sanofi, said: “With three pivotal late-stage trials, our research has been focused on delivering a first-in-class respiratory syncytial virus prevention for all infants.

Our Phase III MELODY results in healthy late preterm and term infants represent a major milestone toward that goal.

We are pleased nirsevimab has the potential to become the first immunization to protect all infants across the respiratory syncytial virus season, with only a single dose.”

A prespecified pooled analysis of the MELODY and Phase IIb trials demonstrated a reduction of hospitalisations caused by RSV with the proposed dose of nirsevimab.

In term and preterm infants (greater than 28 weeks gestational age), 21 of 786 (2.7%) infants in the placebo arm compared to nine of 1,564 (0.6%) in the nirsevimab arm experienced an RSV-associated hospitalisation, giving an estimate of efficacy of 77.3% (95% CI 50.3, 89.7; P<0.001) through 150 days postdose.

There was numerical reduction, although not statistically significant, of the risk of RSV associated hospitalisations observed in the MELODY trial alone (62.1%, 95% CI: -8.6, 86.8, p=0.07)

 In the nirsevimab arm, six of 994 (0.6%) infants were hospitalised for RSV LRTI, while eight of 496 infants (1.6)% were hospitalised in the placebo arm.

The overall safety profile of nirsevimab remains consistent with previously reported results.

No clinically meaningful differences in safety results between the nirsevimab and placebo groups were seen in MELODY and Phase IIb.

MELODY trial results were published in the New England Journal of Medicine (NEJM).

Details from the MEDLEY trial were also published in the NEJM. Regulatory submissions began in the first half of 2022.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/nirsevimab-significantly-protected-infants-against-rsv-disease-in-phase-iii-melody-trial.html

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