Rare disease drugmaker files for approval despite objections from FDA on adequate trial data

Zachary Brennan

Senior Editor

Another small biotech attacking a super-rare disease is leveraging a patient community and very limited clinical data to try to win an FDA approval. …

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Rare disease drugmaker files for approval despite objections from FDA on adequate trial data

Zachary Brennan

Senior Editor

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Sign up to read this article for free.

Trump names Johns Hop­kins re­searcher Mar­ty Makary to lead the FDA; al­so names CDC, Sur­geon Gen­er­al picks

FDA ap­proves Bridge­Bio's heart dis­ease drug, set­ting up com­pe­ti­tion with Pfiz­er, Al­ny­lam

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Rare disease drugmaker files for approval despite objections from FDA on adequate trial data

Trump names Johns Hopkins researcher Marty Makary to lead the FDA; also names CDC, Surgeon General picks

FDA approves BridgeBio's heart disease drug, setting up competition with Pfizer, Alnylam

Drug or biologic? An Eli Lilly lawsuit could impact weight loss drug compounders

Surrounded by AI bio giants, tiny startup Nabla Bio makes de novo antibodies look more like drugs

RFK Jr. and Elon Musk want to ban pharma ads. They wouldn't be the first to try

Are programs with compounded GLP-1s safe for your population?