Timeline of the gene therapy’s clinical development and regulatory review shows agency staff repeatedly questioned the ability of micro-dystrophin to predict clinical benefit in Duchenne muscular dystrophy patients.
Throughout Sarepta Therapeutics, Inc.’s clinical development of the gene therapy Elevidys (delandistrogene moxeparvovec-rokl, formerly SRP-9001), US Food and Drug Administration review staff repeatedly questioned the ability of micro-dystrophin, a surrogate endpoint, to predict clinical benefit in Duchenne muscular dystrophy patients.
Review documents show that in at least four separate meetings with Sarepta dating back to December 2018, Center for Biologics...
The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.
The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.
Manufacturers likely will have to determine when to provide a 340B discount on a drug assigned a Medicare negotiated price under CMS guidance. PhRMA anticipates the job will not be easy.
<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>
