Once again, the FDA is delaying BioMarin’s hemophilia A quest as gene therapy frets simmer
Two years after the FDA turned back BioMarin’s push for a landmark accelerated approval of their hemophilia A gene therapy valrox, regulators are asking for still more info on the therapy — and once again delaying any prospective launch, this time into next year.
Buried under the latest set of extended data from their Phase I/II trial of valrox (valoctocogene roxaparvovec), which continues to demonstrate its effectiveness in preventing bleeds up to 6 years after initial treatment, the biotech spelled out the reasons why they’re shoving back an expected resubmission from June to September. The agency wants more data and analysis, says the biotech, but isn’t asking for any new studies. And the news will likely enhance some deep-seated concerns that the FDA appears to be in no rush to approve any gene therapy overshadowed by questions of durability and safety.
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