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EMA receives marketing authorisation application for molnupiravir for COVID 19

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EMA receives marketing authorisation application for molnupiravir for COVID 19

November 23, 2021: “EMA has started evaluating an application for marketing authorisation for the oral antiviral medicine Lagevrio (molnupiravir). Lagevrio, which is being developed by Merck Sharp & Dohme in collaboration with Ridgeback Biotherapeutics, is intended for the treatment of COVID-19 in adults. 

EMA will assess the benefits and risks of Lagevrio under a reduced timeline and could issue an opinion within weeks if the data submitted are sufficiently robust and complete to show the efficacy, safety and quality of the medicine. 

Such a short timeframe is only possible because EMA has already reviewed a substantial portion of the data on the medicine during a rolling review.

During this phase, EMA’s human medicines committee (CHMP) assessed data from laboratory and animal studies (non-clinical data), information on the quality of the medicine and the way it will be produced, and data on its efficacy and safety.

In addition, CHMP assessed data from completed and ongoing clinical studies. These include interim results from the main study on the effects of Lagevrio in non-hospitalised, unvaccinated patients with at least one underlying condition putting them at risk of severe COVID-19.

Furthermore, EMA’s committee for medicines for children (PDCO) has issued its opinion on the company’s paediatric investigation plan (PIP), which describes how the medicine should be developed and studied for use in children, in accordance with the accelerated timelines for COVID-19 products.

If EMA concludes that the benefits of Lagevrio outweigh its risks in treating COVID‑19, it will recommend granting a marketing authorisation.

The European Commission will then fast-track its decision-making process with a view to granting a marketing authorisation valid in all EU and EEA Member States within days.

EMA will communicate at the time of CHMP’s opinion. 

How is the medicine expected to work?

Lagevrio is an oral antiviral medicine that reduces the ability of SARS-CoV-2 (the virus that causes COVID-19) to multiply in the body.

It does so by introducing alterations (mutations) in the genetic material (known as RNA) of SARS-CoV-2 during replication in a way that impairs the ability of the virus to multiply. This is expected to reduce the need for hospitalising patients with COVID-19.”

https://www.ema.europa.eu/en/news/ema-receives-application-marketing-authorisation-lagevrio-molnupiravir-treating-patients-covid-19

Merck to Present at the 4th Annual Evercore ISI HealthCONx Conference

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Merck to Present at the 4th Annual Evercore ISI HealthCONx Conference

November 24, 2021: “Merck known as MSD outside the United States and Canada, announced today that Frank Clyburn, executive vice president and president of Human Health, is scheduled to participate in a virtual fireside chat at the 4th Annual Evercore ISI HealthCONx Conference on December 1, 2021 at 10:30 a.m. ET.

Investors, analysts, members of the media and the general public are invited to watch a live video webcast of the presentations at https://investors.merck.com/events-and-presentations/default.aspx.”

https://www.merck.com/news/merck-to-present-at-the-4th-annual-evercore-isi-healthconx-conference/

University of Glasgow reveals drug design for new Alzheimer’s treatments

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University of Glasgow reveals drug design for new Alzheimer’s treatments

November 24, 2021: “An international team of scientists and pharmaceutical collaborators have made a breakthrough ‘bench to bedside’ discovery, ten years in the making, which they hope will advance the future treatment of Alzheimer’s Disease in patients.

The research – conducted at the University of Glasgow and the biotechnology company Sosei-Heptares Ltd – and published in Cell describes, for the first time, the process of designing a new molecule to selectively target a specific receptor protein in the brain and demonstrating, through laboratory preclinical and human clinical studies, the potential of this approach to create superior new drugs to improve cognitive function in Alzheimer’s Disease patients.

There are currently no drugs that can stop or slow the progression of Alzheimer’s Disease.

However, there are drugs that work to recover memory loss and improve cognitive function in early dementia, but these drugs are often not very effective, and are associated with side effects that may limit their effectiveness in clinical practice.

The study was focused on new molecules, designed by Sosei Heptares, that selectively target a protein called the M1 muscarinic acetylcholine receptor (or M1 receptor, a G protein-coupled receptor or GPCR) in the brain, which is known to play a central role in memory and cognition.

Subsequent translational medicine studies tested the hypothesis that such molecules will retain cognitive benefits and lack dose-limiting side effects.

Working closely together, the research team demonstrated that an exquisitely selective modulator could be successfully designed using detailed knowledge of the M1 receptor’s 3D structure, despite its very close similarity to other types of muscarinic receptor.

This approach is known as structure-based drug design or SBDD.

Subsequent pre-clinical studies confirmed the designed molecule retained optimal properties for improving memory while minimising side effects associated with previous attempts to target the M1 receptor.

Finally, clinical studies demonstrated that the M1-selective clinical candidate HTL9936, at meaningful doses in healthy volunteers, showed greatly reduced side effects relative to many non-selective predecessors from programmes spanning a generation of traditional drug discovery approaches.

These ground-breaking results substantiate the hypothesis and the power of novel approaches using 3D structures of receptors developed by the team can be applied to the M1 receptor to create a potential new medicine for Alzheimer’s Disease patients, and also have broader implications highlighting a new approach to address other GPCRs linked to a wide range of diseases.

The approach described in the Cell paper has since been used to design other new molecules with superior properties for treating the symptoms of Alzheimer’s Disease and other dementias, and advance these in preclinical development.

Prof. Andrew Tobin, Professor of Molecular Pharmacology and Director of the newly built Advanced Research Centre at the University of Glasgow, said: “This is a true bench-to-bedside discovery, many years in the making, and we are thrilled that this hugely important and global collaboration with our partners at Sosei Heptares and others has resulted in a highly sophisticated drug design approach that offers huge potential to improve the treatment of Alzheimer’s Disease, by activating memory and cognitive centres within the brain.

“We are extremely encouraged by our findings so far and are very hopeful that this could lead to new treatment options for patients with this devastating disease.”

Dr Miles Congreve, Chief Scientific Officer at Sosei Heptares, added: “We are delighted that this pioneering drug design work in collaboration with Prof. Tobin and others has been published in such a highly acclaimed journal. The precision SBDD technology that we have developed is core to our approach at Sosei Heptares and has been extremely effective for designing new and superior drug molecules that we are progressing through preclinical and clinical development as potential new treatments for a wide range of neurological, immunological and gastrointestinal diseases.”

Dr Malcolm Weir, Executive Vice Chairman of Sosei Heptares, added: “We are delighted to be able to report this important advance in the field of new medicines for cognitive disorders such as Alzheimer’s Disease.

It exemplifies our core vision of applying SBDD to create new drugs against challenging targets and taking them through pre-clinical and clinical studies to answer key translational medicine questions, and simultaneously to meet major value inflections.”

Professor Tobin’s work, based at the Centre for Translational Pharmacology at the University of Glasgow’s Institute of Molecular, Cell and Systems Biology, is supported by a £4.2M Wellcome Collaborative Award – funding awarded in 2016, in collaboration with researchers at Monash University in Melbourne Australia (Professors Arthur Christopoulos and Patrick Sexton), to use state-of-the-art technologies and approaches to discover new ways to make better drugs for neurological diseases including Alzheimer’s disease.”

https://www.gla.ac.uk/news/headline_820111_en.html

EC grant Trodelvy marketing authorisation for the treatment of breast cancer

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EC grant Trodelvy marketing authorisation for the treatment of breast cancer

November 23, 2021: “Gilead Sciences, Inc. announced that the European Commission (EC) has granted marketing authorization for Trodelvy® (sacituzumab govitecan), a first-in-class Trop-2-directed antibody-drug conjugate, as a monotherapy indicated for the treatment of adult patients with unresectable or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for advanced disease.

“The metastatic stage of TNBC is particularly challenging to treat and until now we have urgently needed new treatment options for people in Europe living with this condition,” said Dr Véronique Diéras, Senior Medical Oncologist Head, Breast Cancer Group, Department of Medical Oncology, Centre Eugène Marquis, Rennes, France.

“Today’s approval including second-line metastatic TNBC is significant for the community as it’s another important step forward in helping women with this disease live longer.”

TNBC is the most aggressive type of breast cancer and accounts for approximately 15% of all breast cancers. It is more frequently diagnosed in younger and premenopausal women and is more prevalent in Black and Hispanic women.

The five-year survival rate for this sub-type of breast cancer is 12%, compared with 28% for other breast cancer types, and these poor outcomes are often coupled with a significant decrease in quality of life, especially in relapsed/refractory disease.

“At Gilead, we push boundaries to deliver transformative science and novel treatment options that address urgent medical needs,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences.

“We understand how difficult metastatic TNBC is to treat and we’re proud that Trodelvy can now offer a second-line treatment option with the potential to bring longer life to people living with this aggressive disease.”

The EC’s decision is supported by results from the Phase 3 ASCENT study, where Trodelvy reduced the risk of death by 49% and improved median overall survival to 11.8 months versus 6.9 months with physician’s choice of chemotherapy (HR: 0.51; 95% CI: 0.41-0.62; p<0.0001).

These data also showed a statistically significant and clinically meaningful 57% reduction in the risk of death or disease worsening and improved median progression free survival (PFS) to 4.8 months from 1.7 months seen with physician’s choice of chemotherapy alone among all randomized patients, which included those with and without brain metastases (HR: 0.43; 95% CI: 0.35-0.54; p<0.0001).

The most common Grade 3 or higher adverse reactions were neutropenia (49.5%), leukopenia (12.0%), diarrhea (10.7%), anemia (10.1%), febrile neutropenia (6.6%), fatigue (5.2%), hypophosphatemia (5.2%), nausea (4.1%) and vomiting (3.0%).

The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.

In addition to this approval, Trodelvy is approved in Australia, Canada, Great Britain, Switzerland, and the United States in metastatic TNBC. Regulatory review is also underway in Singapore and China with applications submitted by Everest Medicines.

Trodelvy was also recently included in the updated ESMO Clinical Practice Guidelines as a preferred treatment option for metastatic TNBC after taxanes.

About the ASCENT Study

The ASCENT study is a global, open-label, randomized Phase 3 study that enrolled more than 500 patients across 230 study locations.

The study evaluated the efficacy and safety of Trodelvy compared with a single-agent chemotherapy of the physician’s choice in patients with unresectable, locally advanced or metastatic TNBC who had received at least two prior systemic treatments.

Patients were randomly allocated to receive either Trodelvy or a chemotherapy chosen by the patient’s treating physician.

The primary endpoint was progression-free survival (PFS, as determined by blinded independent central review) in patients without brain metastases. Secondary endpoints included: PFS for full study population or intention-to-treat (ITT) population, overall survival in both the ITT population and in the subgroup without brain metastasis, independently determined objective response rate, duration of response, time to onset of response according to Response Evaluation Criteria in Solid Tumors (RECIST 1.1), quality of life and safety.

More information about ASCENT is available at http://clinicaltrials.gov/show/NCT02574455.

About Triple-Negative Breast Cancer (TNBC)

TNBC is the most aggressive type of breast cancer and accounts for approximately 15% of all breast cancers.

TNBC is diagnosed more frequently in younger and premenopausal women and is more prevalent in Black and Hispanic women.

TNBC cells do not have estrogen and progesterone receptors and have limited human epidermal growth factor receptor 2 (HER2).

Due to the nature of TNBC, effective treatment options are extremely limited compared with other breast cancer types. TNBC has a higher chance of recurrence and metastases than other breast cancer types.

The average time to metastatic recurrence for TNBC is approximately 2.6 years compared with 5 years for other breast cancers, and the relative five-year survival rate is much lower. Among women with metastatic TNBC, the five-year survival rate is 12%, compared with 28% for those with other types of metastatic breast cancer.

About Trodelvy

Trodelvy is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein overexpressed in multiple types of epithelial tumors, including metastatic TNBC and metastatic urothelial cancer (UC), where high expression is associated with poor survival and relapse.

Trodelvy is approved in second-line metastatic TNBC in multiple countries worldwide, including Australia, Canada, Great Britain, the European Union, Switzerland and the United States. Trodelvy is also approved for use in metastatic UC in the United States.

Trodelvy continues to be developed for potential use in other TNBC and metastatic UC populations and is also being developed as an investigational treatment for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer and metastatic non-small cell lung cancer.

Additional evaluation across multiple solid tumors is also underway.”

https://www.gilead.com/news-and-press/press-room/press-releases/2021/11/trodelvy-sacituzumab-govitecan-granted-european-commission-marketing-authorization-for-treatment-of-metastatic-triplenegative-breast-cancer-in-sec

EU approves Roche’s Gavreto for RET fusion-positive advanced non-small cell lung cancer

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EU approves Roche’s Gavreto for RET fusion-positive advanced non-small cell lung cancer

November 19, 2021: “Roche announced that the European Commission (EC) has granted conditional marketing authorisation for Gavreto® (pralsetinib) as a monotherapy for the treatment of adults with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.

Gavreto is the first and only precision medicine approved in the European Union (EU) for the first-line treatment of people with RET fusion-positive advanced NSCLC.

“Today’s approval represents an important step forward in delivering precision medicine to people with RET fusion-positive advanced non-small cell lung cancer, for whom treatment options have historically been limited,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development.

“By using cancer genomic profiling upfront, healthcare professionals may identify specific genetic alterations that predict clinical benefit of targeted treatment options like Gavreto in the first-line setting.”

The approval is based on results of the ongoing phase I/II ARROW study, in which Gavreto led to durable responses in people with advanced RET fusion-positive NSCLC.

In 75 treatment-naïve patients, Gavreto demonstrated an overall response rate (ORR) of 72.0% (95% CI: 60.4%, 81.8%), and median duration of response (DOR) was not reached (NR) (95% CI: 9.0 months, NR). 

In 136 patients who had previously received platinum-based chemotherapy, Gavreto demonstrated an ORR of 58.8% (95% CI: 50.1%, 67.2%), and median DOR was 22.3 months (95% CI: 15.1 months, NR).

Gavreto was also generally well-tolerated, with a low rate of treatment discontinuation; common grade 3-4 adverse reactions were neutropenia (reported in 20.1% of patients), anaemia (17.6%) and hypertension (16.1%).

Approximately 37,500 people are diagnosed with RET fusion-positive NSCLC worldwide each year; the disease often affects people with minimal to no history of smoking, and who are typically younger than the average person diagnosed with lung cancer.

Roche is committed to providing a tailored treatment option for every person with lung cancer, no matter how rare or difficult-to-treat their type of disease.

Gavreto in RET fusion-positive advanced NSCLC, along with Alecensa® (alectinib) in ALK-positive advanced NSCLC and Rozlytrek® (entrectinib) in ROS1-positive advanced NSCLC, is part of Roche’s growing portfolio of precision medicines.

Together, they offer personalised treatment options for almost one in ten people with advanced NSCLC, and biomarker testing is the most effective way to identify those people who may benefit.

Beyond NSCLC, RET alterations are also key disease drivers in other cancer types, such as thyroid cancers.

Gavreto has shown activity across multiple solid tumour types, reflecting tumour-agnostic potential.

It is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with metastatic RET fusion-positive NSCLC, and for the treatment of adult and paediatric patients 12 years of age and older with advanced RET-altered thyroid cancers.

Gavreto is also approved in Canada, mainland China and Switzerland. In the EU, a submission for RET-altered thyroid cancers is planned.

Regulatory submissions for advanced RET fusion-positive NSCLC and RET-altered thyroid cancers are also underway in multiple countries worldwide.

Blueprint Medicines and Roche are co-developing Gavreto globally, with the exception of certain territories in Asia, including China.*

Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, are commercialising Gavreto in the US and Roche has exclusive commercialisation rights for Gavreto outside of the US, with the exception of certain territories in Asia, including China.

About the ARROW study
ARROW is an ongoing phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer, RET fusion-positive thyroid cancer and other RET-altered solid tumours.

ARROW is being conducted at multiple sites across the United States, Europe and Asia.

About rearranged during transfection (RET)-altered cancers
RET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including non-small cell lung cancer (NSCLC) and several types of thyroid cancer.

There are approximately 2.21 million cases of lung cancer diagnosed each year worldwide, of which approximately 1.8 million are NSCLC and RET fusions are present in approximately 1-2% of these patients, meaning RET fusion-positive NSCLC affects up to 37,500 people each year.

Additionally, approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumours, and roughly 90% of people with advanced medullary thyroid cancer (a less prevalent form of thyroid cancer) carry RET mutations.

Oncogenic RET fusions also are observed at low frequencies in other cancers, including cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.

About Gavreto® (pralsetinib)
Gavreto is a once-daily, oral precision medicine designed to selectively target rearranged during transfection (RET) alterations, including fusions and mutations, regardless of the tissue of origin.

Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment.

Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.”

https://www.roche.com/media/releases/med-cor-2021-11-19.htm

NICE creates new menu of treatment options for those suffering from depression

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NICE creates new menu of treatment options for those suffering from depression

November 23, 2021: “An independent NICE guideline committee has developed the first guideline for 12 years to identify, treat and manage depression in adults.

It has looked at the evidence on the treatment of new depressive episodes, chronic depression, preventing relapse, patient choice, and the organisation of, and access to, mental health services.

The committee has created a menu of treatment options to allow patients to pick the one which is right for them, in a shared decision-making discussion between them and their healthcare practitioner.

Patients with less severe depression could choose from the menu as a first-line treatment option from, for example, cognitive behavioural therapy (CBT), exercise, counselling or psychotherapy.

A similar range of psychological interventions, along with the option of antidepressant medication, is available to those choosing a first-line treatment for more severe depression.

Dr Paul Chrisp, director of the centre for guidelines at NICE, said: “People with depression deserve and expect the best treatment from the NHS which is why this guideline is urgently required.

“The COVID-19 pandemic has shown us the impact depression has had on the nation’s mental health. People with depression need these evidence-based guideline recommendations available to the NHS, without delay.”

According to the Office of National Statistics, around one in six (17%) adults aged 16 years and over in Great Britain experienced some form of depression in summer 2021.

The rate remains higher than those observed before the coronavirus pandemic (July 2019 to March 2020), where 10% of adults experienced some form of depression.

Nav Kapur, professor of psychiatry and population health at the University of Manchester and chair of the guideline committee, said: “This is a broad ranging guideline on depression which has been an enormous challenge to produce. 

“In particular we’ve emphasised the role of patient choice – suggesting that practitioners should offer people a choice of evidence-based treatments and understanding that not every treatment will suit every person.

We now need stakeholders’ help to make the recommendations as good as they can possibly be.” 

Retired solicitor Catherine Ruane, a lay member on the guideline committee who acted as a carer to two family members with depression, said: “There has been significant progress in science and medicine in the past 12 years.

This guideline emphasises a greater amount of patient choice and takes greater account of the things that really matter to the patients and their carers.”

The guideline also contains new recommendations for those stopping antidepressant medication.

Public Health England’s 2019 evidence review, ‘Dependence and withdrawal associated with some prescribed medicines‘, showed 17% of the adult population in England (7.3 million people) had been prescribed antidepressants in the year 2017 to 2018.

People who are considering taking, or stopping, antidepressants medication should talk with their healthcare professional about the benefits and risks.

The healthcare professional should explain that withdrawal may take weeks or months to complete successfully, that it is usually necessary to reduce the dose in stages over time (called ‘tapering’) and that most people stop antidepressants successfully.”

https://www.nice.org.uk/news/nice-creates-new-menu-of-treatment-options-for-those-suffering-from-depression

FDA Approves First Treatment for Common Type of Post-Transplant Infection that is Resistant to Other Drugs

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FDA Approves First Treatment for Common Type of Post-Transplant Infection that is Resistant to Other Drugs

November 23, 2021: “The U.S. Food and Drug Administration approved Livtencity (maribavir) as the first drug for treating adults and pediatric patients (12 years of age and older and weighing at least 35 kilograms) with post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral treatment for CMV. Livtencity works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication.

“Transplant recipients are at a much greater risk for complications and death when faced with a cytomegalovirus infection,” said John Farley, M.D., M.P.H., director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research.

“Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern.

Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.” 

CMV is a type of herpes virus that commonly causes infection in patients after a stem cell or organ transplant.

CMV infection can lead to CMV disease and have a major negative impact on transplant recipients, including loss of the transplanted organ and death. 

Livtencity’s safety and efficacy were evaluated in a Phase 3, multicenter, open-label, active-controlled trial that compared Livtencity with a treatment assigned by a researcher running the study, which could include one or two of the following antivirals used to treat CMV: ganciclovir, valganciclovir, foscarnet or cidofovir.

In the study, 352 transplant recipients with CMV infections who did not respond (with or without resistance) to treatment randomly received Livtencity or treatment assigned by a researcher for up to eight weeks. 

The study compared the two groups’ plasma CMV DNA concentration levels at the end of the study’s eighth week, with efficacy defined as having a level below what is measurable. Of the 235 patients who received Livtencity, 56% had levels of CMV DNA below what was measurable versus 24% of the 117 patients who received an investigator-assigned treatment.

The most common side effects of Livtencity include taste disturbance, nausea, diarrhea, vomiting and fatigue. Livtencity may reduce the antiviral activity of ganciclovir and valganciclovir, so coadministration with these drugs is not recommended.

Virologic failure due to resistance can occur during and after treatment with Livtencity, therefore CMV DNA levels should be monitored and Livtencity resistance should be checked if the patient is not responding to treatment or relapses.

Livtencity received Breakthrough Therapy and Priority Review designations for this indication.

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.

The FDA granted the approval of Livtencity to Takeda Pharmaceuticals Company Limited.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-common-type-post-transplant-infection-resistant-other-drugs

AstraZeneca unveils The Discovery Centre (DISC) in Cambridge

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AstraZeneca unveils The Discovery Centre (DISC) in Cambridge

November 23, 2021: “AstraZeneca will today, in the presence of His Royal Highness The Prince of Wales, formally unveil The Discovery Centre (DISC) in Cambridge, UK – a state-of-the-art research and development (R&D) facility designed to the world’s highest environmental standards and accommodating over 2,200 research scientists.

The new £1bn facility will include the most advanced robotics, high-throughput screening and AI-driven technology.

It will support AstraZeneca’s focus on specialised and precision medicines and foster the discovery and development of next generation therapeutics, including nucleotide-based, gene-editing and cell therapies.

Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “Our ambition today is to not only unveil a building, but to also drive the next wave of scientific innovation.

Our new Discovery Centre in Cambridge raises the bar for sustainable R&D and global collaboration across our industry.

It will allow us to break new boundaries in the understanding of disease biology, bring life-changing medicines to patients and power the next stage of our company’s growth.”

The DISC will add to AstraZeneca’s R&D presence in more than 40 countries across the globe, including its other strategic research centres in Sweden and the US, and development facilities in China and Japan.

The Company invests more than $7bn in R&D globally each year, a large part of which takes place in the UK. The Centre will help further nurture partnerships, develop the next generation of science leaders and accelerate AstraZeneca’s industry-leading levels of productivity.

Located within the Cambridge Biomedical Campus, the physical proximity of the building’s 19,000m2 laboratories to leading hospitals, the University of Cambridge, other research institutions and a number of biotech companies will promote a culture of open partnership and innovation in its inviting open spaces.

The Company has over 200 active collaborations in the region and more than 2,000 around the world across academia, biotech and industry.

During the unveiling today, His Royal Highness will meet the Chief Executive, the Chairman, Leif Johansson, and other senior leaders, to undertake a walking tour of the new facility and make a short address to invited guests.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/astrazeneca-unveils-the-discovery-centre-disc-in-cambridge.html

FDA Expands Eligibility for COVID-19 Vaccine Boosters

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FDA Expands Eligibility for COVID-19 Vaccine Boosters

November 19, 2021: “The U.S. Food and Drug Administration amended the emergency use authorizations (EUA) for both the Moderna and Pfizer-BioNTech COVID-19 vaccines authorizing use of a single booster dose for all individuals 18 years of age and older after completion of primary vaccination with any FDA-authorized or approved COVID-19 vaccine.

The Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices will meet later today to discuss further clinical recommendations.

“Throughout the course of the COVID-19 pandemic, the FDA has worked to make timely public health decisions as the pandemic evolves.

COVID-19 vaccines have proven to be the best and highly effective defense against COVID-19.

Authorizing the use of a single booster dose of either the Moderna or Pfizer-BioNTech COVID-19 vaccine for individuals 18 years of age and older helps to provide continued protection against COVID-19, including the serious consequences that can occur, such as hospitalization and death,” said Acting FDA Commissioner Janet Woodcock, M.D.

Prior to today’s authorizations, a single booster dose of the Moderna and Pfizer-BioNTech COVID-19 vaccines was authorized for administration to individuals 65 years of age and older, individuals 18 through 64 years of age at high risk of severe COVID-19 and individuals 18 through 64 years of age with frequent institutional or occupational exposure to SARS-CoV-2.

Today’s action expands the use of booster doses of both vaccines to include all individuals 18 years of age and older at least six months after completion of the primary vaccination series of the Moderna COVID-19 Vaccine or Pfizer-BioNTech COVID-19 Vaccine or at least two months after completion of primary vaccination with the Janssen COVID-19 Vaccine.

“The FDA has determined that the currently available data support expanding the eligibility of a single booster dose of the Moderna and Pfizer-BioNTech COVID-19 vaccines to individuals 18 years of age and older,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

“Streamlining the eligibility criteria and making booster doses available to all individuals 18 years of age and older will also help to eliminate confusion about who may receive a booster dose and ensure booster doses are available to all who may need one.”

Data Supporting Effectiveness

The EUA for a single booster dose for individuals 18 years of age and older for the Moderna (administered as half of the dose of a primary series dose) and Pfizer-BioNTech COVID-19 vaccines is based on the FDA’s analysis of immune response data that supported use in the previously authorized populations for boosters. 

For the Moderna COVID-19 Vaccine booster dose, the FDA analyzed the immune response data from 149 participants 18 years of age and older from the original clinical studies who received a booster dose at least six months after their second dose and compared it to the immune responses of 1,055 study participants after completing their two-dose series.

The antibody response against the SARS-CoV-2 virus 29 days after a booster dose of the vaccine demonstrated a booster response. 

For the Pfizer-BioNTech COVID-19 Vaccine booster dose, the FDA analyzed the immune response data from approximately 200 participants 18 through 55 years of age who received a single booster dose about six months after their second dose.

The antibody response against the SARS-CoV-2 virus one month after a booster dose of the vaccine when compared to the response one month after the two-dose primary series in the same individuals demonstrated a booster response.

FDA Evaluation of Benefits and Risks

Since Moderna and Pfizer-BioNTech initially submitted safety and effectiveness data on a single booster dose following primary vaccination to the FDA, additional real-world data have become available on the recently increasing number of cases of COVID-19 in the U.S. and on the risk of myocarditis (inflammation of the heart muscle) and pericarditis (inflammation of the outer lining of the heart) following vaccination with these vaccines.

These additional data enabled the FDA to reassess the benefits and risks of the use of these vaccines in the general adult population.

The FDA has determined that the benefits of a single booster dose of either the Moderna or Pfizer-BioNTech COVID-19 vaccines outweigh the risks of myocarditis and pericarditis in individuals age 18 years of age and older when used following completion of primary vaccination to provide continued protection against COVID-19 and the associated serious consequences that can occur including hospitalization and death.

Both Pfizer and Moderna are conducting post-authorization/post-marketing studies to assess known serious risks of myocarditis and pericarditis.

In addition, the FDA and the CDC have several systems in place to continually monitor COVID-19 vaccine safety and allow for the rapid detection and investigation of potential safety concerns.

The fact sheets for both vaccines for recipients and caregivers and for healthcare providers contain information about the potential side effects, including the risk of myocarditis and pericarditis.

The most commonly reported side effects by individuals who received a booster dose of the vaccines were pain, redness and swelling at the injection site, as well as fatigue, headache, muscle or joint pain and chills.

Of note, swollen lymph nodes in the underarm were observed more frequently following the booster dose than after the primary two-dose series. 

The FDA did not hold a meeting of the Vaccines and Related Biological Products Advisory Committee on these actions as the agency previously convened the committee for extensive discussions regarding the use of booster doses of COVID-19 vaccines and, after review of both Pfizer’s and Moderna’s EUA requests, the FDA concluded that the requests do not raise questions that would benefit from additional discussion by committee members.

The amendments to the EUAs were granted to ModernaTX Inc. and Pfizer Inc.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-expands-eligibility-covid-19-vaccine-boosters

FDA Issues Final Orders Reclassifying Certain Hepatitis C Diagnostic Tests from Class III to Class II

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FDA Issues Final Orders Reclassifying Certain Hepatitis C Diagnostic Tests from Class III to Class II

November 19, 2021: “The following quote is attributed to Timothy Stenzel, M.D., Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in FDA’s Center for Devices and Radiological Health:“Today’s action allows manufacturers of certain types of Hepatitis C virus (HCV) tests to seek marketing clearance through the less burdensome premarket notification (510(k)) pathway rather than submitting a premarket approval application (PMA), the most stringent type of FDA medical device review.

We are confident that following reclassification, with adherence to the special controls, these devices will continue to provide a reasonable assurance of safety and effectiveness.

Additionally, the reclassification may support the potential for more manufacturers to develop these tests, which can increase competition and increase access to these important tests.

These reclassifications will also benefit the Department of Health and Human Services’ National Viral Hepatitis Action Plan, as increased access to tests will likely aid patients in seeking the appropriate treatment and likely reduce transmission.”
 

Additional Information

  • The FDA issued two final orders, reclassifying certain HCV diagnostic tests from class III to II. These orders allow these HCV tests to use FDA’s 510(k) pathway rather than the PMA pathway.
  • The two types of HCV diagnostic tests being reclassified are nucleic acid-based HCV ribonucleic acid (RNA) devices intended for the qualitative or quantitative detection or genotyping of HCV RNA and certain HCV antibody devices intended for the qualitative detection of HCV.”

https://www.fda.gov/news-events/press-announcements/fda-brief-fda-issues-final-orders-reclassifying-certain-hepatitis-c-diagnostic-tests-class-iii-class

FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism

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FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism

November 19, 2021: “The U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow.

Achondroplasia is the most common form of dwarfism. 

“Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States and underscores the FDA’s commitment to help make new therapies available for rare diseases,” said Theresa Kehoe, M.D., director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research.

“With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.” 

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth.

The average height of an adult with achondroplasia is approximately four feet.

People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth.

Voxzogo works by binding to a specific receptor called natriuretic peptide receptor-B that reduces the growth regulation gene’s activity and stimulates bone growth. 

Voxzogo’s safety and efficacy in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase 3 study in participants five years and older with achondroplasia who have open epiphyses.

In the study, 121 participants were randomly assigned to receive either Voxzogo injections under the skin or a placebo. Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year.

Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo. 

The most common side effects of Voxzogo include injection site reactions, vomiting and decreased blood pressure. Voxzogo’s labeling also lists decreased blood pressure as a warning and precaution, which means it is a potentially serious side effect.

The FDA approved Voxzogo under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need, based on a surrogate or intermediate clinical endpoint.

A condition of this accelerated approval is a post-marketing study that will assess final adult height. This application also received priority review designation.

The FDA granted the approval of Voxzogo to BioMarin.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-improve-growth-children-most-common-form-dwarfism

Bristol Myers Squibb Announces New PDUFA Date for Mavacamten

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Bristol Myers Squibb Announces New PDUFA Date for Mavacamten

Nov 11, 2021: “Bristol Myers Squibb announced that the U.S. Food and Drug Administration has extended the review of the NDA for mavacamten for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to April 28, 2022.

The FDA notified Bristol Myers Squibb on November 18, 2021 of the extension of the PDUFA date to allow sufficient time to review information pertaining to updates to the proposed Risk Evaluation Mitigation Strategy (REMS).

A REMS program was included in the initial application for mavacamten. No additional data or studies have been requested.

“We are confident in the profile of mavacamten.

This first-in-class cardiac myosin inhibitor demonstrated clinically meaningful improvements in symptoms, functional status, and quality of life in symptomatic oHCM patients in the pivotal EXPLORER-HCM trial,” said Samit Hirawat, M.D., executive vice president, chief medical officer, global drug development, Bristol Myers Squibb.”

“We look forward to continuing to work closely with the FDA to bring this important medicine to patients.”

https://news.bms.com/news/corporate-financial/2021/Bristol-Myers-Squibb-Announces-New-PDUFA-Date-for-Mavacamten/default.aspx

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