FDA clinical, clinical pharmacology and statistical review teams did not recommend approval of Elevidys, but the CBER director concluded that randomized data in a subgroup of patients ages four and five years old were ‘compelling,’ justifying accelerated approval; expansion beyond this age-restricted subgroup will hinge on data from the EMBARK confirmatory trial.
Sarepta Therapeutics, Inc.’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) won US Food and Drug Administration accelerated approval in an age-restricted population of Duchenne muscular dystrophy patients thanks to Center for Biologics Evaluation and Research Director Peter Marks, who overruled objections from various review disciplines.
The FDA granted accelerated approval to Elevidys (formerly known as SRP-9001) on 22 June. The AAV-directed gene therapy is indicated...
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
<p>Executive Summary<p> <p>The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add six new products, incl/ding Ixchiq, Valneva's chikungunya virus vaccine</p>
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
<p>Executive Summary</p> <p>Zanidatamab, Jazz and BeiGene’s investigational treatment for biliary tract cancer, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.</p>
<p>Executive Summary</p> This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
