A rare disease drug is rejected, even as the FDA talks about new approval pathway

Taking stock of the drug industry, from the lab to the medicine chest

May 29, 2025 Check out STAT+

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By Ed Silverman Pharmalot Columnist, Senior Writer

Adobe STAT+ | A rare disease drug is rejected, even as the FDA talks about new approval pathway The FDA withheld its endorsement of a drug for Barth syndrome, but decided it may be possible for Stealth BioTherapeutics to pursue accelerated approval. By Ed Silverman STAT+ | As the FDA and NIH eschew animal testing, startup raises $40 million for lab-grown organs FDA, NIH push to reduce or replace animal testing expected to spur growth in organoid firms; Dan Huh's Vivodyne announces $40 million investment. By Allison DeAngelis Post-TIGIT flop, iTeos Therapeutics is shuttering Today's biotech news includes more biopharma trials happening only in China, Merck and Daiichi Sankyo withdrawing an FDA application, etc. By Meghana Keshavan

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