FDA agrees to speed review for an ultra-rare disease drug

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

August 21, 2025 Check out STAT+

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By Ed Silverman Pharmalot Columnist, Senior Writer

Adobe STAT+ | FDA agrees to speed review for an ultra-rare disease drug Stealth BioTherapeutics has experienced a roller-coaster ride attempting to convince the FDA to approve its Barth syndrome therapy. By Ed Silverman STAT+ | Major U.S. drug manufacturing plant did not properly investigate cat hair, pests, and other problems, FDA finds The Indiana plant, once owned by Catalent and now owned by Novo Nordisk, is widely used by the pharmaceutical industry. By Ed Silverman and Elaine Chen STAT+ | As Sarepta Therapeutics fights for Duchenne therapy, a group of patients gets left behind After deaths and FDA setbacks, Sarepta abandoned LGMD gene therapy, leaving families desperate and patients without long-promised treatments. By Jason Mast Kite Pharma STAT+ | Gilead's Kite Pharma acquires Interius BioTherapeutics for $350 million The purchase by Kite is latest move in a race to develop simpler, safer, and more scalable cell therapies. By Jason Mast STAT+ | FDA 'uncertainty' forces Krystal Biotech to shutter cancer drug trial  The decision follows the agency's rejection of a similar drug developed by Replimune Group earlier this month. By Adam Feuerstein

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