The FDA expects to issue a decision in the first quarter of 2025, and if the agency grants approval, it will expand the biomarker-eligible population for the drug.

In a draft guidance, the institute did not recommend Orserdu, but is collecting additional data and public comments on this preliminary decision.

The clinical trial testing ATA-200 to treat a type of LGMD caused by mutations in the SGCG gene has already been cleared in Italy and France.

Patients had fewer bleeds after treatment with Beqvez compared to standard treatment, but questions remain about which ...