After ‘conflicting’ signals from FDA, rare disease drugmaker contemplates closure

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

August 5, 2025 Check out STAT+

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By Ed Silverman Pharmalot Columnist, Senior Writer

Adobe STAT+ | After 'conflicting' signals from FDA, rare disease drugmaker contemplates closure The company has so far failed to convince the agency to reconsider its recent rejection of a drug for Barth syndrome, an ultra-rare disease. By Ed Silverman Opinion: Americans should have access to drugs approved overseas Congress should enact reciprocal approval for drugs and medical devices cleared by comparable countries. By Jeffrey Singer and Bautista Vivanco STAT+ | Vertex next-generation pain candidate fails Phase 2 trial The results for VX-993, a NaV1.8 inhibitor, were one of several setbacks for Vertex's next-generation pain program. By Jason Mast and Jonathan Wosen

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