Two sickle cell therapies among FDA’s 13 PDUFA dates in December
Decisions also include bids to convert the accelerated approvals of Amgen’s Lumakras and Calliditas’ Tarpeyo
FDA has at least 13 target action dates coming up before year-end, including applications for two gene therapies that could bring functional cures to patients with sickle cell disease.
CRISPR Therapeutics AG (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) have a Dec. 8 PDUFA date for sickle cell therapy Casgevy exagamglogene autotemcel (exa-cel), which the U.K’s MHRA approved on Nov. 16., making it the world’s first CRISPR-based gene therapy to receive a regulatory approval. The consensus at FDA’s advisory committee meeting last month was that the risks posed by off-target edits amounted to a small factor in the broader evaluation of exa-cel, which uses CRISPR to modify patients’ cells ex vivo, restoring fetal hemoglobin expression. The key recommendation from the meeting was a 15-year patient follow-up period to identify any clinical consequences that may emerge from off-target edits. ...