Regulatory
The HHS Office of Inspector General found that of 278 accelerated approvals between 1992 and 2021, 104 had incomplete confirmatory trials, according to a report issued Thursday.
Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.
The FDA ordered Avidity Biosciences to halt clinical trial enrollment after a patient suffered a “serious adverse event” testing the company’s candidate drug for myotonic dystrophy type 1.
The FDA has granted a rolling review to CRISPR Therapeutics and Vertex’s exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
Daiichi Sankyo won approval in Japan for Ezharmia, making it the first dual inhibitor of EZH1 and EZH2 to receive regulatory approval for ATL.
In an 8-4 vote, the FDA’s Oncologic Drugs Advisory Committee Roster voted against the benefit-risk profile of Secura Bio’s P13K inhibitor Copiktra (duvelisib).
The FDA might only have two PDUFA dates on the calendar this week, but all eyes in the neurodegenerative disease community will be on the first one: Amylyx’s AMX0035 for ALS.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 14-2 against Oncopeptides’ Pepaxto, a drug that has been on the market since 2021 for multiple myeloma under accelerated approval.
The FDA will meet Friday to discuss the future of Secura Bio’s Copiktra (duvelisib), which has been approved as a third-line treatment for relapsed or refractory CLL/SLL.
The U.S. FDA had a busy week with a range of drug approvals, advisory committee meetings and classification announcements.
PRESS RELEASES