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Novartis presents positive Phase III results for Cosentyx® in children with active ERA and JPsA at ACR 2021

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Novartis presents positive Phase III results for Cosentyx® in children with active ERA and JPsA at ACR 2021

November 08, 2021: “Novartis, a leader in rheumatology and immuno-dermatology, today announced new analyses from the two-year positive Phase III JUNIPERA study, which demonstrated the treatment response of Cosentyx® (secukinumab) in children and adolescents with enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA) – two categories of juvenile idiopathic arthritis (JIA).

The safety profile was consistent with that of Cosentyx in adults with plaque psoriasis, psoriatic arthritis, non-radiographic axial spondyloarthritis and ankylosing spondylitis.

These data will be highlighted as an oral presentation at the American College of Rheumatology (ACR) Convergence all-virtual annual meeting, November 3–10, 2021.

Initial results from the Phase III JUNIPERA trial were previously presented at the European Alliance of Associations for Rheumatology (EULAR) 2021 European Congress of Rheumatology in June.

Two-year results from JUNIPERA demonstrated that patients treated with Cosentyx had a significantly longer time to flare, showing a 72% reduction in the risk of flare (P<.001) versus placebo, in children and adolescents ages two to 17 years old with active ERA (n=52; mean age: 13.7) and active JPsA (n=34; mean age: 12.2).

At Week one, over 30% of patients showed improvement with Cosentyx (JIA American College of Rheumatology [ACR] 30) and nearly 90% achieved JIA ACR 30 by the end of the first treatment period (12 weeks).

Additionally, by Week 12, nearly 35% of patients (N=86) achieved JIA ACR inactive disease status.

Improvements in disease activity, as measured by the mean juvenile arthritis disease activity score (JADAS 27), were observed at Week one, reaching low disease activity from Week 12 through Week 104.

Safety in this pediatric population was consistent with the known safety profile of Cosentyx.  

“If left untreated, ERA and JPsA can have a substantial negative impact on quality of life and may lead to deformities and long-term disability for children and adolescents who live with these conditions,” said Dr. Hermine Brunner, Cincinnati Children’s Hospital Medical Center and lead investigator of the JUNIPERA study.

“It is promising that the JUNIPERA study shows that secukinumab demonstrated marked responses in patients with ERA and JPsA, a population that currently has limited treatment options available to help improve joint inflammation, dactylitis and enthesitis.”

Novartis has filed regulatory submissions for Cosentyx in ERA and JPsA in Europe and the US. Final decisions by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) are anticipated in the coming months.

If approved, Cosentyx would be the first biologic treatment for children living with ERA in the US.

“With more than 500,000 patients treated worldwide since launch, these JUNIPERA data further reinforce the known efficacy and safety of Cosentyx for children and adults living with rheumatologic and dermatologic conditions,” said Todd Fox, Global Head of Medical Affairs Immunology, Hepatology and Dermatology at Novartis.

“We are committed to bringing Cosentyx to young people living with inflammatory rheumatic diseases as quickly as possible, as part of our ambition to expand Cosentyx to 10 indications.”

In July 2020, Cosentyx received EU approval as a first-line systemic treatment for pediatric psoriasis and recently received approval in the US and Chin.

In Japan, Cosentyx has also been approved to treat psoriatic arthritis as well as psoriasis in pediatric patients aged 6 years or older, as well as generalized pustular psoriasis.

About the JUNIPERA Study
JUNIPERA is a two-year, three-part, double-blind, placebo-controlled, randomized-withdrawal, Phase III study that enrolled 86 children and adolescents aged 2 to 17 years with a confirmed diagnosis of JPsA or ERA according to the International League of Associations for Rheumatology classification criteria.

Patients were given open-label secukinumab 75 mg/150 mg (prefilled syringe at doses of 75 mg in patients <50 kg and 150 mg in patients ≥50 kg) up until Week 12.

In this treatment period 1, patients achieving at least JIA ACR 30 response then progressed onto treatment period 2 where patients were allocated to one of two arms: secukinumab 75 mg/150 mg (depending on bodyweight) or placebo and responses observed up until Week 104.

The primary endpoint of the study was time to flare in the treatment period 2 (Week 12 to Week 104).

Secondary endpoints in treatment period 1 (up to Week 12) included evaluation of JIA ACR 30/50/70/90/100 responses and each JIA ACR core component, change from baseline of the JADAS, and total enthesitis and dactylitis count.

About Cosentyx® (secukinumab)
Cosentyx is the first and only fully-human biologic that directly inhibits interleukin-17A (IL-17A), an important cytokine involved in the inflammation of psoriatic arthritis, (PsA), moderate to severe plaque psoriasis, (PsO), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA).

Cosentyx is a proven medicine and has been studied clinically for more than 14 years. The medicine is backed by robust evidence, including five years of clinical data in adults supporting long-term safety and efficacy across moderate to severe PsO, PsA and AS.

These data strengthen the position of Cosentyx as a treatment across AS and nr-axSpA, PsA and moderate to severe PsO, supported by more than 500,000 patients treated worldwide since launch in 2015.

https://www.novartis.com/news/media-releases/novartis-presents-positive-phase-iii-results-cosentyx-children-active-enthesitis-related-arthritis-era-and-juvenile-psoriatic-arthritis-jpsa-acr-2021

Takeda applied for selection to “Prime Market” of Tokyo Stock Exchange

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Takeda applied for selection to “Prime Market” of Tokyo Stock Exchange

November 1, 2021: “Takeda Pharmaceutical Company Limited applied for selection of the “Prime Market” segment of the Tokyo Stock Exchange, Inc. (“TSE”) as its listing market as determined at the Board of Directors Meeting held on September 29 and 30, 2021.

Takeda received the results of the initial assessment made by the TSE on July 9, 2021, confirming Takeda’s compliance with the listing requirements for the “Prime Market” in the new market segments that will be introduced on April 4, 2022 by the TSE.

In addition, Takeda today filed its Corporate Governance Report with the TSE based on the Corporate Governance Code, which was revised on June 11, 2021.

This report describes the status of compliance with the disclosure items required of companies listed on the “Prime Market.”

“As one of the largest R&D-driven biopharmaceutical companies in the world, and the only one listed on both the TSE and NYSE, our ambition is to build a world-class governance model to lead the company into the future” said Christophe Weber, Representative Director, President and CEO of Takeda.

Takeda  also enhanced the Corporate Governance page of its website to improve the transparency of the company’s corporate governance and to promote understanding among its shareholders and investors.

This includes publication of the company’s Articles of Incorporation, Board of Directors Charter, Nomination Committee Charter, Compensation Committee Charter, Audit and Supervisory Committee Charter, and Company Share Policy.

The Corporate Governance Report and the corporate governance-related documents, can be found on the company’s corporate website at https://www.takeda.com/who-we-are/corporate-governance/.

 “Being listed on the TSE’s ‘Prime Market’ will enable us to maximize our corporate value through increased transparency as well as further enhance our engagement with stakeholders including our shareholders and investors in Japan and around the world,” added Koki Sato, Corporate Strategy Officer and Chief of Staff of Takeda.”

https://www.takeda.com/newsroom/newsreleases/2021/Takeda-Applied-for-Selection-of-Prime-Market-Filed-Corporate-Governance-Report-with-Tokyo-Stock-Exchange-TSE-in-Line-with-Revised-Corporate-Governance-Code-and-Published-Corporate-Governance-related-Documents-on-its-website/

AZ to transfer global rights for Eklira and Duaklir to Covis Pharma

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AZ to transfer global rights for Eklira and Duaklir to Covis Pharma

 November 01, 2021: “AstraZeneca has agreed to transfer its global rights to Eklira (aclidinium bromide), known as Tudorza in the US, and Duaklir (aclidinium bromide/formoterol) to Covis Pharma Group (Covis Pharma).

Both medicines are delivered via the Genuair device and used for the treatment of patients with chronic obstructive pulmonary disease (COPD).

The agreement will ensure continued patient access to these established medicines.

Covis Pharma previously acquired the rights to the respiratory medicines Alvesco, Omnaris and Zetonna from AstraZeneca in 2018.

Financial considerations
Covis Pharma will pay AstraZeneca $270m on completion. Covis Pharma will also cover certain ongoing development costs related to the medicines.

The income arising from the upfront payment will be fully offset by a charge for derecognition of the associated intangible asset and therefore no Other Operating Income will be recognised in AstraZeneca’s financial statements.

In 2020, Eklira and Duaklir generated AstraZeneca revenue of $143m in the countries covered by this agreement.

The transaction is expected to close in the fourth quarter of 2021, subject to customary closing conditions and regulatory clearances.

The agreement will not impact the Company’s financial guidance for 2021.

Eklira and Duaklir
Eklira (aclidinium bromide) and Duaklir (aclidinium bromide/formoterol) are inhaled respiratory medicines used for the maintenance treatment of COPD.

Eklira is a long-acting muscarinic antagonist (LAMA), which is marketed in the US as Tudorza and in some countries as Bretaris. 

Duaklir is a combination therapy that contains both a LAMA and a long-acting beta2-agonist (LABA).

It is marketed in some countries as Brimica. Both medicines are presented as a dry powder for inhalation and are delivered via a breath-actuated multi-dose dry powder inhaler, Genuair (Pressair in the US).

AstraZeneca licensed the global rights to both products from Almirall S.A. in 2014.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/astrazeneca-to-transfer-rights-to-eklira-duaklir.html

FDA approves Novartis Scemblix® for chronic myeloid leukemia

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FDA approves Novartis Scemblix® for chronic myeloid leukemia

October 29, 2021: “Novartis announced that the US FDA approved Scemblix® (asciminib) for the treatment of chronic myeloid leukemia (CML) in two distinct indications.

The FDA granted Scemblix accelerated approval for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs), based on major molecular response (MMR) rate at 24 weeks; and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

In accordance with the Accelerated Approval Program, continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence.

Scemblix is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket, and represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies.

Also known as a STAMP inhibitor in scientific literature, Scemblix is being studied across multiple treatment lines for CML-CP, including the ASC4FIRST Phase III study evaluating Scemblix as a first-line treatment.

“The introduction of TKIs twenty years ago revolutionized treatment for CML; however, there remain many patients who do not respond adequately to at least two available treatments and often experience challenging side effects that add a burden to their daily lives,” said Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society.

“The approval of Scemblix may offer hope to patients by addressing gaps in CML care.”

For many patients, current treatment for CML may be limited by intolerance or resistance, and sequential use of available TKIs is associated with increased failure rates.

In an analysis of patients with CML treated with two prior TKIs, approximately 55% reported intolerance to previous treatment.

Additionally, a pooled analysis in the second-line setting showed that up to 70% of patients are unable to achieve major molecular response (MMR) within two years of follow-up.

Moreover, patients who develop the T315I mutation are resistant to most available TKIs, leaving them at an increased risk of disease progression.

“CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both,” expressed Dr. Michael J. Mauro**, Hematologist and Myeloproliferative Neoplasms Program Leader at Memorial Sloan Kettering Cancer Center (MSK).

“The addition of Scemblix into the CML treatment landscape gives us a novel approach to combat this blood cancer, helping address clinical challenges in patients struggling after switching to a second treatment, as well as in patients who develop the T315I mutation and face significantly worse outcomes.”

The FDA approval of Scemblix is based on results from the Phase III ASCEMBL trial and a Phase I (NCT02081378) study that included patients with Ph+ CML-CP with the T315I mutation.

In patients with Ph+ CML-CP who had experienced resistance or intolerance to at least two TKIs, the ASCEMBL trial showed that1-3:

  • Scemblix nearly doubled the MMR rate vs. Bosulif® (bosutinib)* at 24 weeks (25% vs. 13% [P=0.029])
  • The proportion of patients who discontinued treatment due to adverse reactions was more than three times lower in the Scemblix arm (n = 156) vs. patients in the Bosulif arm (n = 76) (7% vs. 25%)
  • The most common (incidence ≥ 20%) adverse reactions and laboratory abnormalities in the Scemblix arm were, respectively: upper respiratory tract infections and musculoskeletal pain; decrease in platelet and neutrophil counts, decrease in hemoglobin; increase in triglycerides, creatine kinase and alanine aminotransferase (ALT)

“After more than two decades of reimagining CML care, we continue to boldly push the boundaries of innovation to transform the standard-of-care and help even more patients living with this disease,” said Susanne Schaffert, PhD, President, Novartis Oncology.

“We would like to thank all those who have been involved in helping to advance this new and important breakthrough.”

Scemblix is currently available for physicians to prescribe to appropriate patients in the US.

Additional efficacy and safety details for Scemblix, including data on patients with the T315I mutation, and full Prescribing Information can be found at https://www.novartis.us/sites/www.novartis.us/files/scemblix.pdf.

About Scemblix® (asciminib)
Scemblix (asciminib) is indicated for the treatment of adult patients with Ph+ CML-CP pre-treated with two or more TKIs, as well as adult patients with Ph+ CML-CP with the T315I mutation.

The first indication is approved under the US FDA Accelerated Approval Program based on MMR rate at 24 weeks; continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence.

Scemblix is the first FDA-approved CML treatment that binds to the ABL myristoyl pocket.

This novel mechanism of action, also known in scientific literature as a STAMP inhibitor, may help address resistance in patients with CML previously treated with two or more TKIs and overcome mutations at the defective BCR-ABL1 gene, which is associated with the over-production of leukemic cells, Scemblix has also been shown to limit off-target activity in pre-clinical studies.

Novartis has initiated regulatory filings for Scemblix in multiple countries and regions across the globe.

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP. 

Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix as a first-line treatment and is in the recruitment phase.

About Novartis Commitment to CML
Novartis has a long-standing scientific commitment to patients living with CML. For more than 20 years, our bold science has helped transform CML into a chronic disease for many patients.

Despite these advancements, we’re not standing still. We continue to research ways to target the disease, seeking to address the challenges with treatment resistance and/or intolerance that many patients face.

Novartis also continues to reimagine CML care through its commitment to sustainable access for patients and collaboration with the global CML community.

Indication
SCEMBLIX® (asciminib) tablets is a prescription medicine used to treat adults with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitor (TKI) medicines.

The effectiveness of SCEMBLIX in these patients is based on a study that measured major molecular response (MMR) rates. No clinical information is available to show if these patients treated with SCEMBLIX live longer or if their symptoms improve.

Ongoing studies exist to find out how SCEMBLIX works over a longer period of time.

SCEMBLIX is also approved for use in adults with Ph+ CML in CP with the T315I mutation.

It is not known if SCEMBLIX is safe and effective in children.”

https://www.novartis.com/news/media-releases/fda-approves-novartis-scemblix-asciminib-novel-mechanism-action-treatment-chronic-myeloid-leukemia

FDA Authorizes Pfizer-BioNTech COVID-19 Vaccine for Emergency Use in Children 5 through 11 Years of Age

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FDA Authorizes Pfizer-BioNTech COVID-19 Vaccine for Emergency Use in Children 5 through 11 Years of Age

October 29, 2021: “The U.S. FDA authorized the emergency use of the Pfizer-BioNTech COVID-19 Vaccine for the prevention of COVID-19 to include children 5 through 11 years of age.

The authorization was based on the FDA’s thorough and transparent evaluation of the data that included input from independent advisory committee experts who overwhelmingly voted in favor of making the vaccine available to children in this age group.

Key points for parents and caregivers:

  • Effectiveness: Immune responses of children 5 through 11 years of age were comparable to those of individuals 16 through 25 years of age. In addition, the vaccine was found to be 90.7% effective in preventing COVID-19 in children 5 through 11.
  • Safety: The vaccine’s safety was studied in approximately 3,100 children age 5 through 11 who received the vaccine and no serious side effects have been detected in the ongoing study.
  • The Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices will meet next week to discuss further clinical recommendations.

“As a mother and a physician, I know that parents, caregivers, school staff, and children have been waiting for today’s authorization.

Vaccinating younger children against COVID-19 will bring us closer to returning to a sense of normalcy,” said Acting FDA Commissioner Janet Woodcock, M.D.

“Our comprehensive and rigorous evaluation of the data pertaining to the vaccine’s safety and effectiveness should help assure parents and guardians that this vaccine meets our high standards.”

The Pfizer-BioNTech COVID-19 Vaccine for children 5 through 11 years of age is administered as a two-dose primary series, 3 weeks apart, but is a lower dose (10 micrograms) than that used for individuals 12 years of age and older (30 micrograms). 

In the U.S., COVID-19 cases in children 5 through 11 years of age make up 39% of cases in individuals younger than 18 years of age.

According to the CDC, approximately 8,300 COVID-19 cases in children 5 through 11 years of age resulted in hospitalization.

As of Oct. 17, 691 deaths from COVID-19 have been reported in the U.S. in individuals less than 18 years of age, with 146 deaths in the 5 through 11 years age group.  

“The FDA is committed to making decisions that are guided by science that the public and healthcare community can trust.  

We are confident in the safety, effectiveness and manufacturing data behind this authorization.

As part of our commitment to transparency around our decision-making, which included our public advisory committee meeting earlier this week, we have posted documents today supporting our decision and additional information detailing our evaluation of the data will be posted soon.

We hope this information helps build confidence of parents who are deciding whether to have their children vaccinated,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

The FDA has determined this Pfizer vaccine has met the criteria for emergency use authorization.

Based on the totality of scientific evidence available, the known and potential benefits of the Pfizer-BioNTech COVID-19 vaccine in individuals down to 5 years of age outweigh the known and potential risks. 

FDA Evaluation of Available Effectiveness Data

The effectiveness data to support the EUA in children down to 5 years of age is based on an ongoing randomized, placebo-controlled study that has enrolled approximately 4,700 children 5 through 11 years of age.  

The study is being conducted in the U.S., Finland, Poland and Spain. Children in the vaccine group received two doses of the Pfizer-BioNTech COVID-19 Vaccine containing 10 micrograms of messenger RNA per dose.

The FDA analyzed data that compared the immune response of 264 participants from this study to 253 participants 16 through 25 years of age who had two higher doses of the vaccine in a previous study which determined the vaccine to be effective in preventing COVID-19.

The immune responses of the younger age participants were comparable to the older participants. 

The FDA also conducted a preliminary analysis of cases of COVID-19 occurring seven days after the second dose.  

In this analysis, among participants without evidence of prior infection with SARS-CoV-2, 3 cases of COVID-19 occurred among 1,305 vaccine recipients and 16 cases of COVID-19 occurred among 663 placebo recipients; the vaccine was 90.7% effective in preventing COVID-19. 

FDA Evaluation of Available Safety Data

The available safety data to support the EUA include more than 4,600 participants (3,100 vaccine, 1,538 placebo) ages 5 through 11 years enrolled in the ongoing study. In this trial, a total of 1,444 vaccine recipients were followed for safety for at least 2 months after the second dose.

Commonly reported side effects in the clinical trial included injection site pain (sore arm), redness and swelling, fatigue, headache, muscle and/or joint pain, chills, fever, swollen lymph nodes, nausea and decreased appetite.

More children reported side effects after the second dose than after the first dose. Side effects were generally mild to moderate in severity and occurred within two days after vaccination, and most went away within one to two days.

The FDA and CDC safety surveillance systems have previously identified increased risks of myocarditis (inflammation of the heart muscle) and pericarditis (inflammation of tissue surrounding the heart) following vaccination with Pfizer-BioNTech COVID-19 Vaccine, particularly following the second dose, and with the observed risk highest in males 12 through 17 years of age.

Therefore, the FDA conducted its own benefit-risk assessment using modelling to predict how many symptomatic COVID-19 cases, hospitalizations, intensive care unit (ICU) admissions and deaths from COVID-19 the vaccine in children 5 through 11 years of age would prevent versus the number of potential myocarditis cases, hospitalizations, ICU admissions and deaths that the vaccine might cause.

The FDA’s model predicts that overall, the benefits of the vaccine would outweigh its risks in children 5 through 11 years of age.

Ongoing Safety Monitoring

Pfizer Inc. has updated its safety monitoring plan to include evaluation of myocarditis, pericarditis and other events of interest in children 5 through 11 years of age.

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 problems.

It is mandatory for Pfizer Inc. and vaccination providers to report to any serious adverse events, cases of Multisystem Inflammatory Syndrome and cases of COVID-19 that result in hospitalization or death in vaccinated individuals.

It is also mandatory for vaccination providers to report all vaccine administration errors to VAERS for which they become aware and for Pfizer Inc. to include a summary and analysis of all identified vaccine administration errors in monthly safety reports to the FDA.

Data Supports New Vaccine Formulation to Improve Stability and Storage Conditions

The FDA today also authorized a manufacturing change for the vaccine to include a formulation that uses a different buffer; buffers help maintain a vaccine’s pH (a measure of how acidic or alkaline a solution is) and stability.

This new formulation is more stable at refrigerated temperatures for longer periods of time, permitting greater flexibility for vaccination providers.

The new formulation of the vaccine developed by Pfizer Inc. contains Tris buffer, a commonly used buffer in a variety of other FDA-approved vaccines and other biologics, including products for use in children.

The FDA evaluated manufacturing data to support the use of Pfizer-BioNTech COVID-19 Vaccine containing Tris buffer and concluded it does not present safety or effectiveness concerns.”

https://www.fda.gov/news-events/press-announcements/fda-authorizes-pfizer-biontech-covid-19-vaccine-emergency-use-children-5-through-11-years-age

EU Approves Myovant Sciences RYEQO for RYEQO® for Treatment of Uterine Fibroids

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EU Approves Myovant Sciences RYEQO for RYEQO® for Treatment of Uterine Fibroids

July 20, 2021: “Myovant Sciences, a healthcare company focused on redefining care for women and for men announced the European Commission (EC) has approved the marketing authorization application for RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age, with no limitation for duration of use. 

The EC decision is valid in all 27 member states of the European Union, as well as Iceland, Norway, and Liechtenstein.

“Data from the Phase 3 LIBERTY program, which supported the approval of RYEQO, showed that RYEQO improved symptoms most relevant to women living with uterine fibroids, namely heavy menstrual bleeding and pain, while maintaining a well-tolerated safety profile,” said Roberta Venturella, M.D., Ph.D., Associate Professor, Magna Græcia University of Catanzaro and investigator in the LIBERTY program.

“With this approval, women and doctors finally have a long-term treatment option, which is important for the management of this condition.”

“Today’s approval of RYEQO, the first and only once-daily long-term treatment for women with uterine fibroids in Europe, marks a major milestone in expanding non-invasive treatment options for this common and potentially debilitating disease,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. 

“Through our partnership with Gedeon Richter, we look forward to supporting even more women suffering from uterine fibroids.”

The approval is based on safety and efficacy data from the Phase 3 LIBERTY program, which consisted of two replicate, 24-week, multinational clinical studies (LIBERTY 1 and LIBERTY 2), a one-year extension study, and supportive bone mineral density data from a randomized withdrawal study.

Results from the LIBERTY 1 and LIBERTY 2 studies were published in the New England Journal of Medicine in February 2021.

In March 2020, Myovant and Gedeon Richter entered into an exclusive license agreement for Gedeon Richter to commercialize relugolix combination tablet for uterine fibroids and endometriosis in Europe, the Commonwealth of Independent States including Russia, Latin America, Australia, and New Zealand.

Under the agreement, Myovant received an upfront payment of $40 million and is eligible to receive up to $40 million in regulatory milestones and $107.5 million in sales milestones for a total of $147.5 million, and tiered royalties on net sales following regulatory approval. 

Gedeon Richter will be responsible for local clinical development, manufacturing, and all commercialization for its territories. 

Myovant and Gedeon Richter will also continue to collaborate on the marketing authorization application for endometriosis, which is expected to be submitted in the second half of calendar year 2021.

About Uterine Fibroids
Uterine fibroids are noncancerous tumors that develop in or on the muscular walls of the uterus and are among the most common reproductive tract tumors in women.

In addition to an individual’s genetic predisposition, estrogens are well known to play an important role in the regulation of fibroid growth.

Although uterine fibroids are benign tumors, they can cause debilitating symptoms such as heavy menstrual bleeding (frequently resulting in anemia and fatigue), pain (including painful periods, abdominal pain, painful intercourse, backache), increased abdominal girth and bloating, urinary frequency or retention, constipation, pregnancy loss, and, in some cases, infertility.

These symptoms can also lead to loss of productivity at work, limitations in normal activities of daily living, and social embarrassment.

About RYEQO®
RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is approved for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age.

RYEQO contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen), which may reduce the risk of bone loss, and norethindrone acetate (a progestin), which is necessary when women with a uterus (womb) take estrogen.”

https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-announces-european-commission-approval-ryeqor/

FDA Strengthens Safety Requirements and Updates Study Results for Breast Implants

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FDA Strengthens Safety Requirements and Updates Study Results for Breast Implants

October 27, 2021: “The U.S. Food and Drug Administration took several new actions to strengthen breast implant risk communication and help those who are considering breast implants make informed decisions.

First, the agency issued orders restricting the sale and distribution of breast implants to help ensure that patients considering breast implants are provided with adequate risk information so that they can make fully informed decisions. Additionally, the agency has approved new labeling for all legally marketed breast implants that includes a boxed warning, a patient decision checklist, updated silicone gel-filled breast implant rupture screening recommendations, a device description with a list of specific materials used in the device and a patient device card.

Finally, the FDA released updated information on the status of breast implant manufacturer post-approval studies.

These actions will help patients understand the risks and benefits of breast implants and make more informed decisions about their health.

“Protecting patients’ health when they are treated with a medical device is our most important priority,” said Binita Ashar, M.D., director of the Office of Surgical and Infection Control Devices in the Center for Devices and Radiological Health.

“In recent years, the FDA has sought more ways to increase patients’ access to clear and understandable information about the benefits and risks of breast implants.

By strengthening the safety requirements for manufacturers, the FDA is working to close information gaps for anyone who may be considering breast implant surgery.

As the FDA continues to evaluate the overall effects of breast implants in patients, today’s actions help ensure that all patients receive the information they need to make well-informed decisions affecting their long-term, personal health.”

The FDA has taken a number of steps to better understand the patient perspective and address risks associated with breast implants, including convening the General and Plastic Surgery Devices Advisory Panel in 2019 to publicly discuss the long-term benefits and risks of breast implants indicated for breast augmentation and reconstruction.

The meeting covered a range of important topics on breast implant safety, including characterization of breast implant associated anaplastic large cell lymphoma (BIA-ALCL) incidence and risk factors and methods for assessing systemic symptoms referred to by patients as breast implant illness.

The panel gave recommendations on these topics, including recommending that the FDA require a boxed warning in breast implant labeling and a standardized checklist as part of the informed consent process, revise the MRI screening recommendations for silent ruptures of silicone gel-filled breast implants and provide greater transparency regarding materials present in breast implants.

Following the 2019 panel meeting, the FDA issued final guidance for breast implants that recommends labeling updates, including a patient decision checklist, screening recommendations for ruptures, information about breast implant materials and a patient device card. The final guidance was issued in September 2020.

The FDA’s order today restricts the sale and distribution of breast implants to only health care providers and facilities that provide information to patients utilizing the patient brochure “Patient Decision Checklist.”

The checklist must be reviewed with the prospective patient by the health care provider to help ensure the patient understands the risks, benefits and other information about the breast implant device.

The patient must be given the opportunity to initial and sign the patient decision checklist and it must be signed by the physician implanting the device.

The FDA is requiring these restrictions based on its finding that the available information indicates such restrictions are necessary to provide a reasonable assurance of the device’s safety and effectiveness.

The FDA is committed to continuing to use its full authorities to ensure the post-market safety of medical products.

The new labeling approved today follows the labeling recommendations described in FDA’s September 2020 guidance and was included as part of the supplemental approval applications submitted by manufacturers.

The FDA orders for IDEAL IMPLANT Structured Saline Breast Implants, Mentor Saline-Filled and Spectrum Breast Implants, Inamed (now Allergan) Natrelle Saline Filled Breast Implants, Inamed (now Allergan) Natrelle Silicone Filled Breast Implants, Mentor MemoryShape Silicone Gel-Filled Breast Implants, Mentor MemoryGel Silicone Gel-Filled Breast Implants and Sientra OPUS Silicone Gel Breast Implants approve the updated labeling in addition to restricting the devices as described above.

Manufacturers are asked to post the updated device labeling to their websites within the next 30 days.

Based on these approval orders, product labeling for these devices now includes a boxed warning to inform patients of significant risks of breast implants, as well as updated silicone gel-filled breast implant rupture screening recommendations, a patient decision checklist, inclusion of a description of materials used in breast implants and chemicals that can be released from breast implants and patient device cards, all of which were recommended at the March 2019 Panel Meeting.

A medical device’s labeling is intended to enhance, but not replace, the physician-patient discussion of the risks and benefits of breast implants that uniquely pertain to individual patients.

The post-approval study data released today furthers the FDA’s commitment to transparently understanding the long-term effects of breast implants by providing status updates of all breast implant studies, as required by the premarket approval (PMA) process.

Breast implants are designated as a Class III medical device, which includes devices that support or sustain human life, are of substantial importance in preventing impairment of human health or which present a potential, unreasonable risk of illness or injury.

Due to the level of risk associated, Class III devices require PMA approval before a manufacturer can legally market their device, which is the most stringent type of device marketing application required by the FDA.”

https://www.fda.gov/news-events/press-announcements/fda-strengthens-safety-requirements-and-updates-study-results-breast-implants

FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases

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FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases

October 27, 2021: ‘The U.S. Food and Drug Administration, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.

While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies.

The newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership (AMP) program and project-managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases.

“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them most,” said Peter Marks, M.D., Ph.D., Director of the Food and Drug Administration (FDA)’s Center for Biologics Evaluation and Research.

“FDA is committed to developing a regulatory paradigm that can advance gene therapies to meet the needs of patients with rare diseases.”

“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” said NIH Director Francis S. Collins, M.D., Ph.D.

“There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.”

A single rare disease affects small numbers of people, but rare diseases collectively affect millions. Most rare inherited diseases stem from a specific gene mutation that is already known, making gene therapy a promising therapeutic approach.

However, gene therapy development for rare diseases is highly complex, time consuming and expensive.  Moreover, the development process is stymied by limited access to tools and technologies, lack of standards across the field, and a one-disease-at-a-time approach to therapeutic development.

A standardized therapeutic development model that includes a common gene delivery technology (a vector) could allow for a more efficient approach to specific gene therapies, saving time and cost. 

“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market,” said Joni L. Rutter, Ph.D., acting director of NIH’s National Center for Advancing Translational Sciences (NCATS).

“The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients.”

A primary aim of BGTC is to improve understanding of the basic biology of a common gene delivery vector known as the adeno-associated virus (AAV).

BGTC researchers will examine the biological and mechanistic steps involved in AAV vector production, vector delivery of genes into human cells and how therapeutic genes are activated in target cells.

These results will provide important information for improving the efficiency of vector manufacturing and enhancing the overall therapeutic benefit of AAV gene therapy. 

To improve and accelerate gene and vector manufacturing and production processes, the BGTC program will develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers.

Such tests could be broadly applicable to different manufacturing methods and make the process of developing gene therapies for very rare conditions much more efficient.

A clinical component of BGTC-funded research will support between four and six clinical trials, each focused on a different rare disease.

These diseases are expected to be rare, single-gene diseases with no gene therapies or commercial programs in development and that already have substantial groundwork in place to rapidly initiate preclinical and clinical studies.

The trials will employ different types of AAV vectors that have been used before in clinical trials. For these trials, the BGTC will aim to shorten the path from studies in animal models of disease to human clinical trials.

The BGTC also will explore methods to streamline regulatory requirements and processes for the FDA approval of safe and effective gene therapies, including developing standardized approaches to preclinical testing (e.g., toxicology studies).

NIH and private partners will contribute approximately $76 million over five years to support BGTC-funded projects.

This includes about $39.5 million from the participating NIH institutes and centers, pending availability of funds.

NCATS, which developed the related Platform Vector Gene Therapy (PaVe-GT) program and is the NIH lead institute for BGTC, expects to contribute approximately $8 million over five years.

Private partners include Biogen Inc., Cambridge, Massachusetts; Janssen Research & Development, LLC, Raritan, New Jersey; Novartis Institutes for BioMedical Research, Cambridge, Massachusetts; Pfizer Inc., New York, New York; REGENXBIO Inc., Rockville, Maryland.; Spark Therapeutics, Philadelphia, Pennsylvania; Takeda Pharmaceutical Company Limited, Deerfield, Illinois; Taysha Gene Therapies, Dallas, Texas; Thermo Fisher Scientific Inc., Waltham, Massachusetts; and Ultragenyx Pharmaceutical, Novato, California. Several non-profit partners also are involved, including the Alliance for Regenerative Medicine (ARM), Washington, D.C.; the American Society of Gene and Cell Therapy, Milwaukee, Wisconsin; CureDuchenne, Newport Beach, California; National Organization for Rare Disorders (NORD), Quincy, Massachusetts; and The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), Newark, Delaware.

In addition to NCATS, participating NIH institutes include the Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Eye Institute; National Heart, Lung, and Blood Institute; National Human Genome Research Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Dental and Craniofacial Research; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; and National Institute on Deafness and Other Communication Disorders. 

The BGTC is the first AMP initiative focused on rare diseases. Other ongoing AMP projects bring together scientific talent and financial resources from academia, industry, philanthropy, and government, and focus on improving the productivity of therapeutic development for common metabolic diseases, schizophrenia, Parkinson’s disease, Alzheimer’s disease, type 2 diabetes and autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus.

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health (FNIH) creates and manages alliances with public and private institutions in support of the mission of the NIH.

The FNIH works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe.

Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit https://fnih.orgExternal Link Disclaimer.

About the National Center for Advancing Translational Sciences (NCATS): NCATS conducts and supports research on the science and operation of translation — the process by which interventions to improve health are developed and implemented — to allow more treatments to get to more patients more quickly.

For more information about how NCATS helps shorten the journey from scientific observation to clinical intervention, visit https://ncats.nih.gov.

About the Food and Drug Administration (FDA): The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.”

https://www.fda.gov/news-events/press-announcements/fda-nih-and-15-private-organizations-join-forces-increase-effective-gene-therapies-rare-diseases

Bayer to speed global development of vegetable seeds varieties at expanded R&D Center in Spain

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Bayer to speed global development of vegetable seeds varieties at expanded R&D Center in Spain

 October 26, 2021: “Bayer signals its commitment to delivering high quality cucumber, melon, pepper and eggplant seeds faster to growers around the world with the announced expansion of its Vegetable Seeds R&D Product Design Center in San Nicolás, Spain.

The San Nicolás site supports Bayer’s global vegetable seeds R&D pipeline for varieties sold to customers in more than 130 countries and territories under the Seminis® and De Ruiter® brands, both united under Vegetables by Bayer.

“By using advanced breeding techniques that help speed up the time to market for new varieties, we’re helping growers respond to unexpected challenges of today and into the future,” said Inci Dannenberg, Head of Global Vegetable Seeds at Bayer.

“Our customers and value chain partners can’t afford to wait around for solutions to new disease pressures, unpredictable weather patterns or rapidly changing market demands.”

With the largest investment in research and development in the industry, Bayer began the €5.1 million expansion of the Product Design Center in September 2019.

The expansion includes a multifunctional R&D facility for cell biology, laboratories, cell culture rooms, climate-controlled chambers, and plans to develop a high-tech greenhouse space.

It also allows for a three-fold increase in capacity for innovative doubled haploid (DH) production methods.

Delivering quality seeds faster for growers, sustainably

DH technology is a valuable tool that shortens breeding cycles, improves consistency in product quality, and can accelerate commercial hybrid seed introductions by up to four years.

The use of dihaploids as part of the breeding process can reduce the time needed to deliver solutions to growers by half.

In addition to developing material sooner for product development and trialing, DH also provides consistency in product quality, a key factor for grower success given the diverse conditions they face around the world.

“What’s really exciting is that we are growing our pipeline without growing our footprint.

Our innovation in plant breeding and cell biology can increase value for growers while decreasing greenhouse gas emissions and reducing environmental impact in both the breeding process and in the field,” said J.D. Rossouw, Head of Bayer’s Vegetables R&D.

“Bayer plans to further invest in San Nicolás over the next few years as part of a second phase expansion, which will enable us to boost DH capabilities nearly six-fold by 2023.”

The San Nicolás site is a key component of a holistic strategy to accelerate the design of novel genetic combinations and produce high quality doubled haploid plants at scale.

This Product Design approach within vegetable breeding enables the incorporation of cutting edge technologies including genomics, cell biology, artificial intelligence, robotics, and imaging to better design products for growers and consumers.

San Nicolás is one of more than 30 best-in-class vegetable R&D sites around the world and one of four Product Design Centers located in key vegetable producing regions including the Netherlands, Guatemala, and California.

Meeting unique and changing consumer preferences

Bayer’s investment in its vegetables R&D pipeline reflects not just the changing needs of growers and the value chain but also those of global and diverse consumers.

As one of the world’s leading producers of vegetable seeds, Bayer provides growers worldwide with best-in-class seeds and solutions addressing consumers’ needs for healthy nutrition and ensuring supply for a stable food value chain.

The San Nicolás announcement comes as the United Nations celebrates the International Year of Fruits and Vegetables in 2021, which aims to raise awareness around ways to promote increased consumption of fruits and vegetables across the value chain.”

https://media.bayer.com/baynews/baynews.nsf/id/Bayer-to-speed-global-development-of-vegetable-seeds-varieties-at-expanded-RD-Center-in-Spain?Open&parent=news-overview-category-search-en&ccm=020

GSK Consumer Healthcare solidifies commitment to pharmacists with 3-year Pharmacist Support Programme

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GSK Consumer Healthcare solidifies commitment to pharmacists with 3-year Pharmacist Support Programme

October 26, 2021: “GSK Consumer Healthcare unveils a three-year programme to support the pharmacy profession with additional practical resources, mental health provisions and proposed policy changes as part of its new report Standing with Pharmacists in the Age of Self-Care.  

The new report summarises findings from a roundtable hosted by GSK Consumer Healthcare in collaboration with the International Pharmaceutical Federation (FIP) earlier this year, which convened the pharmacy community, policy experts, and industry leaders to discuss the critical role of pharmacy in the age of self-care following the COVID-19 pandemic.

“We’ve seen COVID-19 have a significant impact on consumers’ understanding of self-care, leading to an increased focus on wellbeing and everyday healthcare.

During this time, pharmacies played a vital role in providing care and advice to their patients – particularly with many other health facilities closed.

Our research shows that more than half of Europeans are planning to consult their pharmacist more often than before the pandemic.

Yet in general, pharmacists remain under-utilised, despite being the most widely distributed health resource in Europe,” said Tess Player, Global Head of Expert at GSK Consumer Healthcare.

The report identifies six barriers faced by the global pharmacy community that can prevent them from fulfilling their critical role.

These are workload; remuneration; limited education on disease prevention; public misperceptions of pharmacists’ expertise; integration challenges within the wider healthcare system; and support for identifying and addressing low health literacy among health consumers.  

To tackle these issues, GSK Consumer Healthcare is unveiling a three-year commitment programme advocating for pharmacy policy and regulation change, and offering a range of bespoke support, training, and tools for pharmacists to help them address the barriers identified in the roundtable.

  • Year 1 (2021-22): Focus will be on providing mental health and wellbeing support for pharmacists and pharmacy teams.

    This will also include the development of behavioural change tools and guidance for pharmacists in self-care conversations.
  • Year 2 (2022-2023): Focus will be on helping pharmacists to manage their workload and wellbeing through the development of patient information and diagnostic tools.

    Year 2 will also include helping pharmacists identify and support health literacy challenges via a health literacy assessment tool.
  • Year 3 (2023-2024): Focus will be on continuing to improve public awareness of the role of pharmacists in self-care among health consumers.

    This will include working with pharmacists to develop resources that improve health literacy, including public awareness initiatives and community health tracking.

“We are proud partners to pharmacists everywhere and are committed to supporting the global pharmacy community to fulfil its critical role in building public health literacy and providing expert care.

Our three-year plan is only the beginning; we have spent years working with experts to gain a better understanding of the barriers the pharmacy community faces and how we can empower them to fulfil their utmost potential,” Tess Player continued.

The programme is part of the company’s long-standing commitment to supporting the pharmacy community.

In September 2020, GSK Consumer Healthcare released a Statement of Intent on the vital role of pharmacists in the future success of healthcare systems worldwide.

“The COVID-19 pandemic accentuated the crucial role pharmacists play in consumers’ self-care routines. Self-care involves a variety of actions that patients take to manage their health.

Pharmacists play an important role in guiding patient’s self-care behaviours. Thorough assessment and effective communication are crucial to meaningful self-care counselling.

Pharmacists can act as advocates who empower patients and help them make healthy lifestyle choices, recommend appropriate OTC medications, and educate consumers about when they should consult a physician,” said Lars-Åke Söderlund, Vice President of the International Pharmaceutical Federation, FIP.

“As the global healthcare system continues to evolve, self-care is expected to have an increasing role in treating certain minor ailments, and pharmacists are at the forefront of these changes and can lead the ‘self-care revolution’”, Söderlund continued.

https://www.gsk.com/en-gb/media/press-releases/gsk-consumer-healthcare-solidifies-commitment-to-pharmacists-with-3-year-pharmacist-support-programme/

CDC Lab Testing Confirms Cause of Melioidosis Outbreak

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CDC Lab Testing Confirms Cause of Melioidosis Outbreak

October 26, 2021: “The Centers for Disease Control and Prevention confirmed that bacteria in a Better Homes & Gardens aromatherapy spray in a Georgia melioidosis patient’s home genetically matches the bacterial strains in the patient, and three other patients in Kansas, Minnesota, and Texas infected since March.

CDC announced Friday that a bottle of the BHG aromatherapy spray in “Lavender & Chamomile with Gemstones” scent had tested positive for Burkholderia pseudomallei, the bacteria that causes melioidosis.

But the final step in the investigation, being reported today, was to confirm the DNA fingerprint of the bacteria in the spray and in the patients was the same. This allows CDC to confirm the spray or one of its ingredients caused the four melioidosis infections. 

“When you think about the thousands of things people come in contact with around their homes, it’s remarkable we were able to identify the source and confirm it in the lab,” said Inger Damon, MD, PhD, director of CDC’s Division of High-Consequence Pathogens and Pathology. “CDC scientists and our partners found the proverbial needle in the haystack.” 

With the Consumer Product Safety Commission (CPSC) and Walmart, CDC has contacted the manufacturer in India to determine whether ingredients from the implicated spray were used in any other products.

CDC scientists are working to assess the extent of contamination in other bottles and whether other scents may also be contaminated. Since Friday, CDC testing of an additional bottle of the spray has tested positive for the bacteria.  

Anyone who has a bottle of BHG Lavender & Chamomile Aromatherapy Spray with Gemstones or other scents in the same product line purchased from Walmart online or in these Walmart storesexternal icon between February and Oct. 21, 2021, should stop using it immediately and follow the steps listed on CDC’s website to safely return the product to Walmart.

CPSC and Walmart recalled about 3,900 bottles of aromatherapy spray on Oct. 22. Walmart is offering consumers a refund and a $20 gift card for its return.  

CDC emphasized that consumers should not pour the contents down the drain or throw the bottle in the trash.

The bacteria that cause melioidosis does not normally live in soil and water in the United States.

If the spray bottles end up in landfills, the bacteria could become established and cause future melioidosis cases in the U.S. CDC is working with Walmart to ensure the returned bottles are disposed of properly and safely.”

https://www.cdc.gov/media/releases/2021/p1026-melioidosis-outbreak.html

Novartis Kymriah® priority review to treat relapsed or refractory follicular lymphoma

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Novartis Kymriah® priority review to treat relapsed or refractory follicular lymphoma

October 27, 2021: “Novartis announced that the US FDA and EMA have accepted the company’s Supplemental Biologics License Application (sBLA) and Type II Variation, respectively, for Kymriah® (tisagenlecleucel) in adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two prior lines of treatment.

The FDA has also granted priority review to the company’s sBLA for Kymriah in adult patients with r/r FL. Kymriah was previously granted orphan medicinal product designation by the European Commission (EC) for FL.

If approved in this potential third indication, Kymriah would have the opportunity to present an important treatment option for those patients with r/r FL in need of potentially definitive outcomes.

The regulatory submissions are based on positive data from the pivotal Phase II ELARA trial, which investigated the efficacy and safety of Kymriah in adult patients with r/r FL.

The trial met the primary endpoint with robust responses observed in heavily pretreated patients.

The safety profile was remarkable, with no patients experiencing grade 3 or higher cytokine release syndrome (CRS) related to Kymriah within the first 8 weeks following infusion.

Data from the trial was presented earlier this year as an oral presentation during the 2021 Annual American Society of Clinical Oncology (ASCO) Virtual Scientific Meeting.

“This is an important milestone in our mission to bring Kymriah to adult patients with relapsed or refractory follicular lymphoma. Receiving orphan drug designation from the EC as well as priority review from the FDA underscores the unmet need and urgency for these patients.

With Kymriah demonstrating impressive results in the ELARA trial, we are hopeful that we can offer a unique and potentially definitive treatment that minimizes the burden,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis.

Orphan drug designation is reserved for medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease with a prevalence in the EU of below 5 in 10,000 and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease.

The decision follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the EMA.

Kymriah also has Orphan Drug designation from the FDA and the Japan Ministry of Health, Labour and Welfare (MHLW) for this disease.

Priority Review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions, as determined by the FDA.

Read related News: Novartis Kymriah® receives FDA Regenerative Medicine Advanced Therapy designation in follicular lymphoma

Novartis updates on BELINDA study for B-cell non-Hodgkin lymphoma


Novartis to unveil new data at ASCO and EHA from its robust portfolio

Kymriah is currently approved by the FDA, EMA and other regulatory authorities for the treatment of r/r pediatric and young adult (up to and including 25 years of age) acute lymphoblastic leukemia (ALL), and r/r adult diffuse large B-cell lymphoma (DLBCL).

About follicular lymphoma
Follicular lymphoma (FL), the second most common form of non-Hodgkin lymphoma (NHL), is an indolent lymphoma, and represents approximately 22% of NHL cases.

It is often an unrelenting malignancy with a relapsing and remitting pattern.

Throughout the lifetime of a patient with relapsing FL, he or she may be exposed to a median of five lines of prior treatment, with an upper range of 13 lines.

Although patients in in third or later line treatment for FL have multiple systemic therapies available, the efficacy of these regimens drops off rapidly in later lines.

Additionally, because of this relapsing and remitting pattern, patients who are refractory to treatment or relapse may exhaust available treatment options.

About the ELARA trial
ELARA is a Phase II, single-arm, multicenter, open-label trial investigating the efficacy and safety of Kymriah in adult patients with r/r FL after at least two prior therapies. This international trial has enrolled patients from over 30 sites in 12 countries worldwide.

The primary endpoint is complete response rate (CRR) based on best response by central review (Lugano 2014 criteria). Patients evaluable for efficacy had measurable disease at infusion and more than six months of follow-up from infusion or discontinued early.

After infusion, disease assessments were performed every three months. Secondary endpoints include overall response rate, duration of response, progression-free survival, overall survival and safety.

Primary analysis data announced at ASCO 2021 showed Kymriah led to responses for the majority of patients treated, with 66% achieving a complete response (95% CI, 56-75). The overall response rate was 86% (95% CI, 78-92).

Importantly, no patients in ELARA trial experienced grade 3 or higher cytokine release syndrome related to Kymriah within the first 8 weeks following infusion, the most common side effect associated with CAR-T therapy.”

https://www.novartis.com/news/media-releases/novartis-receives-priority-review-us-fda-and-filing-acceptance-ema-kymriah-treat-patients-relapsed-or-refractory-follicular-lymphoma

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