BioMarin plans to file for approval of its investigational hemophilia A gene therapy in the EU in June this year but its US filing is not planned until Q2 2022. It will be the company's second attempt at success in both jurisdictions.
BioMarin Pharmaceutical's Roctavian (valoctocogene roxaparvovec), the company's investigational gene therapy for adults with severe hemophilia A, will be fast-tracked through the EU medicines evaluation system once the company files for approval. BioMarin plans to submit a marketing authorization application to the European Medicines Agency in June.
An accelerated assessment at the EMA can shave a few months off the year or so it usually takes for...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
