STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications
The Trump administration on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients’ individual mutations.
Food and Drug Administration Commissioner Marty Makary and biologics chief Vinay Prasad had already previewed the new approach, known as the plausible mechanism pathway, in a New England Journal of Medicine article in November. But Monday’s rules are step one for actually turning the idea into policy and will provide crucial details for groups hoping to develop such individualized medicines.
The new pathway has been greeted enthusiastically by academics, companies, and patient groups who see it as the only way to usher in gene editing-based treatments and other drugs for patients with unique mutations or mutations too rare to interest pharma.
Jason Mast and Lizzy Lawrence
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