Tag Archive for: FDA

Sarepta Therapeutics said on Friday that the U.S. Food and Drug Administration would review an application seeking traditional approval for its gene therapy to treat a muscle-wasting disorder by June 21, months after it failed the main goal of a confirmatory trial.

In late 2023, Vertex Pharmaceuticals and CRISPR Therapeutics made history by gaining the first FDA approval for a CRISPR-based drug, exagamglogene autotemcel (Casgevy) for the treatment of sickle cell disease with vaso-occlusive crisis. This historic achievement coincided with a record number of 14 review designations awarded by the FDA to CRISPR-based therapies in 2023, according to GlobalData.

According to the FDA, Lilly’s Mounjaro is available only in limited quantities through early March 2024.

After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.

The company announced that it will not sell new devices to treat sleep apnea in the United States in the coming years as it works to comply with a settlement with the Food and Drug Administration.

The agency approved the use of Regeneron and Sanofi’s Dupixent to treat an allergic inflammation of the esophagus in children aged one to 11 years old and weighing at least 15 kg.

The FDA’s Peter Marks and Nicole Verdun in the NEJM Wednesday disclosed that more than 27,000 CAR-T doses have been administered in the U.S. as of the end of 2023, of which there are 22 cases of T-cell cancers.

The FDA was expected to make its decision by Wednesday, but it is still reviewing Liquidia’s marketing application for possible approval, although it has not asked for any additional clinical data to support the application.

The regulator’s Complete Response Letter flagged issues with the new formulation of tesamorelin’s chemistry, manufacturing and controls and sought more information about its immunogenicity risk.

At the beginning of January, the FDA announced that it was authorizing Florida’s Agency for Health Care Administration to import certain prescription drugs from Canada. Leslie Isenegger, practice leader, corporate pricing and public affairs at Real Chemistry, answers some key questions about the implications the program could have on the pharma industry.