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Déjà Vu: CBER Director’s Intervention Saves Sarepta’s Elevidys Again

 
• By Bridget Silverman

Failure of the EMBARK confirmatory trial convinced US FDA reviewers the Duchenne muscular dystrophy gene therapy did not show clinical benefit, but biologics center director Peter Marks invoked totality of evidence to grant full approval.

Boy in wheelchair
FDA expanded use of Elevidys to non-ambulatory DMD patients under accelerated approval. • Source: Shutterstock

The US Food and Drug Administration’s full approval of Sarepta Therapeutics, Inc.’s Elevidys (delandistrogene moxeparvovec-rokl) pastes another jewel in the company’s “most contentious approvals” crown, with Center for Biologics Evaluation and Research Director Peter Marks again stepping in to over-rule the recommendations of the review team.

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New EU Approvals

 
• By Neena Brizmohun

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.

New EU Filings

 
• By Neena Brizmohun

Levodopa/carbidopa (ND0612), Mitsubishi Tanabe Pharma’s investigational drug-device combination therapy for the treatment of motor fluctuations in people with Parkinson’s disease, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Japan Recommends Beyonttra, Raises Enhertu Price

 
• By Lisa Takagi

Japan recommends eight new drugs for approval, including two for cardiomyopathy, and will also raise prices for multiple products, including Enhertu, on 1 April.

Generic Approvals Maintained Pace In December And January, But Is A Slow-Down Coming?

 
• By Derrick Gingery

Unofficial January results showed the US FDA continued to issue full and tentative ANDA approvals in line with previous months, but recent layoffs may not help the agency maintain that pace.

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EMA Considers Whether Intrathecal Zolgensma Deserves Fast Tracking

 
• By Neena Brizmohun

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.

Sponsors Of Two Orphans And An Acne Treatment To Defend Marketing Authorization Applications At EMA

 
• By Francesca Bruce

Sponsors of three drugs that are in the final stages of the EU regulatory review cycle are due to make the case for marketing approval before the European Medicines Agency.

EU Marketing Approval Gap Between Lilly’s Donamemab and Eisai’s Leqembi Could Narrow

 
• By Francesca Bruce

The European Medicines Agency is this week set to issue an opinion on whether pan-EU marketing should be granted to Lilly’s Alzheimer’s disease drug donanemab.