Peter Marks, who headed the FDA’s Center for Biologics Evaluation and Research before being forced to resign in March, said the agency’s new risk-based COVID-19 vaccine framework contradicts the ...

Galapagos named former Horizon Therapeutics exec Aaron Cox as CFO. Saatchi & Saatchi Wellness, a 2025 MM+M Agency 100 honoree, named Brendan Gallagher as president, Beth Bour as chief client officer ...

FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...

Sarepta stock dropped again Wednesday after the FDA announced it's investigating the deaths of two patients who received Elevidys.

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Compass Pathways' Phase 3 COMP-360 data met primary endpoints but disappointed vs analyst expectations. See why investors ...

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

The agency said it will evaluate whether further regulatory action is needed after investigating the deaths of two patients ...

Capricor's DMD therapy Deramiocel is under FDA Priority Review with no major issues flagged; PDUFA date set for August 31, 2025.

Vinay Prasad—the controversial head of vaccine regulation at the US Food and Drug Administration (FDA)—has been named FDA chief medical and scientific officer.1 Traditionally separate roles, his new ...

Good morning! Today, we get deeper into the ousting of Nicole Verdun at the FDA, see promise from Vertex’s cell therapy for ...

Speaking at BIO2025, rare disease leaders from Ultragenyx, Amylyx and Yale questioned the need for the new regulatory pathway ...