May 29, 2025 07:14 AM EDT

FDA rejects Stealth's rare disease drug, but offers potential for accelerated approval

Zachary Brennan

Senior Editor

After a slight delay, the FDA rejected Stealth BioTherapeutics’ Barth syndrome drug application for elamipretide, the company said early Thursday, but will consider a resubmission …

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FDA rejects Stealth's rare disease drug, but offers potential for accelerated approval

Zachary Brennan

Senior Editor

Sign up to read this article for free.

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FDA re­jects Stealth's rare dis­ease drug, but of­fers po­ten­tial for ac­cel­er­at­ed ap­proval

Zachary Brennan

Senior Editor

Sign up to read this article for free.

Supreme Court to keep mulling state PBM dis­pute as US urges jus­tices to de­cline case

Large phar­ma com­pa­nies spell out rea­sons why tar­iffs will harm US in­vest­ments

Mer­ck, Dai­ichi Sankyo scrap FDA ap­pli­ca­tion for HER3 ADC af­ter sur­vival miss

DNA gi­ant Il­lu­mi­na spawned an ecosys­tem of com­peti­tors and cus­tomers

In­tel­lia re­ports case of liv­er tox­i­c­i­ty in AT­TR gene edit­ing study

From weeks to days: how Aizon In­tel­li­gent Agents trans­form phar­ma val­i­da­tion

FDA rejects Stealth's rare disease drug, but offers potential for accelerated approval

Supreme Court to keep mulling state PBM dispute as US urges justices to decline case

Large pharma companies spell out reasons why tariffs will harm US investments

Merck, Daiichi Sankyo scrap FDA application for HER3 ADC after survival miss

DNA giant Illumina spawned an ecosystem of competitors and customers

Intellia reports case of liver toxicity in ATTR gene editing study

From weeks to days: how Aizon Intelligent Agents transform pharma validation