The first hemophilia gene therapy has a date with the FDA
The FDA has promised to rule on BioMarin’s one-time treatment for hemophilia A by Aug. 21. And if everything goes according to the company’s plan, it could open an insurmountable lead on its many competitors.
The agency accepted BioMarin’s application for the gene therapy valrox, granting it a priority review and, at least for now, deciding not to convene a panel of experts before deciding whether to approve it.
In the meantime, BioMarin has amassed enough capacity to manufacture about 10,000 commercial-grade doses of gene therapy each year. That means valrox could treat every eligible hemophilia A patient in the U.S. within about two years of winning approval, according to the company.
That could be trouble for Roche and Sangamo Therapeutics, which are more than a year behind BioMarin with hemophilia A gene therapies of their own. But it depends first on BioMarin convincing the FDA, and then on the all-important matter of how much these companies decide to charge for their products.
The agency accepted BioMarin’s application for the gene therapy valrox, granting it a priority review and, at least for now, deciding not to convene a panel of experts before deciding whether to approve it.
In the meantime, BioMarin has amassed enough capacity to manufacture about 10,000 commercial-grade doses of gene therapy each year. That means valrox could treat every eligible hemophilia A patient in the U.S. within about two years of winning approval, according to the company.
That could be trouble for Roche and Sangamo Therapeutics, which are more than a year behind BioMarin with hemophilia A gene therapies of their own. But it depends first on BioMarin convincing the FDA, and then on the all-important matter of how much these companies decide to charge for their products.
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