Bluebird Bio Inc (BLUE)

[stocksrising]

FYI

bluebird bio Submits Biologics License Application (BLA) to FDA for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events.

The severity of sickle cell disease, and its impact on patients and caregivers, has been underappreciated and overlooked for far too long. Transformative therapies for this community are long overdue," said Andrew Obenshain, chief executive officer, bluebird bio. "We are pleased to have satisfied the Agency's questions about comparability to enable our BLA submission, and to take this important step toward making lovo-cel available for individuals living with SCD."

Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease. The BLA submission is based on efficacy results from 36 patients in the HGB-206 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.

The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for the treatment of SCD.
Plus a new 13g filing last week :)