STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications\u00a0
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The Trump administration\u00a0on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients\u2019 individual mutations.<\/p>\n
Food and Drug Administration\u00a0Commissioner 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<\/a> in November. But Monday\u2019s rules are step one for actually turning the idea into policy and will provide crucial details for groups hoping to develop such individualized medicines.\u00a0<\/p>\n 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.\u00a0<\/p>\n Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n