bluebird: AdComm Will Not Be Scheduled for lovo-cel Gene Therapy for Sickle Cell Disease

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel). Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) with a proposed indication for patients ages 12 and older who have a history of vaso-occlusive events (VOEs). The Agency previously accepted the lovo-cel Biologics Licensing Application (BLA) for Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.

"Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease and represents the third lentiviral vector gene therapy that the Agency has reviewed from bluebird—giving us great confidence in the robustness and maturity of our BLA package," said Andrew Obenshain, chief executive officer, bluebird bio. "We remain focused on working with the Agency on its review in anticipation of a decision by the end of this year."

The BLA for lovo-cel is based on efficacy results from 36 patients in the HGB-206 study Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each. The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up, which is the longest follow-up of any gene therapy program for SCD.

The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.

https://finance.yahoo.com/news/bluebird-bio-confirms-fda-communicated-110000837.html