Regulatory
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.
The FDA granted rare pediatric disease designation to AVR-RD-04, an experimental gene therapy treatment for the lysosomal storage disease cystinosis, AVROBIO announced Tuesday.
Shares of Spectrum Pharmaceuticals plunged 33% upon the release of briefing documents for an FDA advisory meeting regarding poziotinib
The FDA has a broad range of activities in the drug and medical device space. Here’s a look at the agency’s work this week.
PRESS RELEASES