FDA Approves BioMarin's Gene Therapy Roctavian for Hemophilia A
TL;DR Summary
The FDA has approved BioMarin's gene therapy, Roctavian, for the treatment of hemophilia A, following a turbulent regulatory journey that included a surprise rejection in 2020 and a review extension earlier this year. BioMarin aims to commercialize Roctavian and hopes it will become a key blockbuster drug, potentially doubling the company's revenues to $4 billion to $5 billion by the middle of the decade. The list price for the therapy has not been disclosed yet.
- BioMarin's hemophilia A gene therapy Roctavian wins FDA approval after delay and rejection Endpoints News
- New Gene Therapy Could Face Uphill Battle The Wall Street Journal
- U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A PR Newswire
- Gene therapy for severe hemophilia is approved by FDA The Hill
- BioMarin wins approval for gene therapy to treat hemophilia A STAT
- View Full Coverage on Google News
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