Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US
Conditional Approval Granted In EU In August
Executive Summary
BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.
You may also be interested in...
ICER Hemophilia Gene Therapy Reviews Show Clear ‘Net Health Benefit’ But Caution On Patient, Payer Fronts Given Upfront Costs
Restrictive interpretations of FDA labels for BioMarin and CSL Behring products by payers could signal slow launch of gene therapies seeking to supplant existing treatments.
BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay
FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.
Finance Watch: With Limited Fundraising Options, Biotechs Restructure To Extend Cash Runways
Mereo will reduce its headcount by 40% to focus on its lead programs while NeuBase revealed a 60% workforce reduction to extend its cash runway. Also, Invivyd (formerly Adagio), Greenlight and BioMarin announced job cuts, while Otonomy and others planned strategic shifts.