bluebird bio: Partial Clinical Hold for Under-18s in Sickle Cell Gene Therapy Program

 bluebird bio, Inc. (NASDAQ: BLUE) today announced that the FDA has placed its clinical program for lovotibeglogene autotemcel (lovo-cel) gene therapy for sickle cell disease (SCD) on partial clinical hold for patients under the age of 18. The partial, temporary suspension relates to an ongoing investigation by bluebird bio into an adolescent patient with persistent, non-transfusion-dependent anemia following treatment with lovo-cel, now 18 months post-treatment. This patient is clinically well and there is no evidence of malignancy or clonal predominance. Enrollment and dosing for patients 18 and older living with SCD in the HGB-206, HGB-210 and LTF-307 clinical studies, as well as follow up for treated patients of all ages in all studies are continuing as planned.

"The safety of patients treated with our gene therapies is always our top priority," said Richard Colvin, MD, Chief Medical Officer, bluebird bio. "Consistent with the FDA’s direction, we have paused enrollment and treatment of patients younger than 18 in our SCD clinical program, and we will continue to work collaboratively with the FDA to understand and address their concerns. While the partial hold is in place, we intend to continue planned follow-up on previously treated patients from HGB-206 and HGB-210 and plan to enroll and treat new adult patients with lovo-cel through study HGB-210 to further characterize the efficacy and safety of lovo-cel for patients with SCD and to continue to advance the field of gene therapy."

Consistent with the FDA’s clinical hold procedures, bluebird anticipates receiving written questions from the agency in early 2022 and will work quickly to respond in order to resolve the partial hold. The company is evaluating what impact, if any, the partial clinical hold may have on first quarter 2023 projected timing for the lovo-cel biologics license application (BLA) submission. As previously communicated, bluebird has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for approval, with the demonstration of analytical comparability and validation of our commercial manufacturing process as the key remaining actions prior to submission of the planned BLA.

https://finance.yahoo.com/news/bluebird-bio-announces-partial-clinical-130000711.html