Beta-Thalassemia Gene Tx Gets Ringing Endorsement From FDA Advisers
An FDA advisory committee on Friday unanimously recommended betibeglogene autotemcel (beti-cel) for approval in patients with transfusion-dependent beta-thalassemia, a chronic inherited blood disorder. With a 13-0 vote, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee said the benefits of the one-time, first-in-class lentiviral vector (LVV) gene therapy outweigh any of the risks associated with the […]
CDC Panel: Thumbs Down on Pfizer Booster for Healthcare Workers
The CDC’s Advisory Committee on Immunization Practices (ACIP) voted Thursday to unanimously recommend a booster dose of Pfizer’s COVID-19 vaccine (Comirnaty) for adults ages 65 and up and those in long-term care facilities, as well as for adults ages 50 to 64 with high-risk medical conditions (by a vote of 13-2), but that was where […]
Safety Concerns Turn FDA Panel Thumbs Down for Novel CKD-Anemia Drug
An FDA advisory committee almost unanimously agreed that the risks were just too high for the chronic kidney disease (CKD)-related anemia drug roxadustat in any patient population. In a 13-1 vote Thursday, members of the Cardiovascular and Renal Drugs Advisory Committee felt that although there’s an unmet need for new anemia therapeutics, this particular drug […]