The U.S. Food and Drug Administration (USFDA) has announced that it has eliminated the Risk Evaluation and Mitigation ...

Restricted availability under an FDA-mandated drug safety program has limited the reach of CAR-T cell therapies for certain blood cancers—until now. | The FDA has removed the REMS requirements for ...

Some of us believe in civil debate and discussion, even though Drs. Vinay Prasad, Marty Makary, and Jay Bhattacharya do not.

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 ...

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

Sarepta stock dropped again Wednesday — extending a steep dive this year — after the Food and Drug Administration announced that it's investigating the deaths of two patients who received Sarepta ...

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

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

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 ...

When Americans imagine the future, it always has a lot to do with flying cars, robot housekeepers, and just about anything ...

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