This week a decision should be made by the FDA on a new gene therapy, betibeglogene autotemcel (beti-cel), Beti-cel is a one-time autologous product designed to treat transfusion-dependent beta thalassemia.
According to bluebird bio, Inc., the maker of beti-cel, there are about 1,300 people in the U.S. with the most severe form of beta thalassemia, but the exact prevalence is not known.(1) Currently the only treatment that may be curative for transfusion-dependent beta thalassemia is allogeneic hematopoietic stem cell transplantation (SCT). However, this is only recommended for younger patients and depends on the availability of an HLA-matched donor. SCT has risks of severe complications such as transplant-related mortality, graft rejection, and graft-versus-host disease; in addition, the recurrence rate has been roughly 72%.(2)
The Institute for Clinical and Economic Review (ICER) judged beti-cel “superior overall to the current standard of care, but the magnitude of that overall net health benefit is less certain, ranging from incremental to substantial (B+).”(3) According to ICER, “traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million with an 80% payback option for patients who do not achieve and maintain transfusion independence over a five-year period.” (4)
The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee reviewed the gene therapy in June 2022 and unanimously voted ‘yes’ (13-0) on the issue of whether the benefits of this treatment outweigh the risks. The final FDA decision is expected on August 19, 2022.
References:
(1) Transfusion Dependent Beta Thalassemia. https://www.bluebirdbio.com/our-focus/transfusion-dependant-beta-thalassemia. Accessed 8/15/2022.
(2)AMS. PredictDx. Thalassemia. Accessed 8/15/2022.
(3)Beaudoin FL, Richardson M, Synnott PG, Lancaster V, Fluetsch N, HerceHagiwara B, Campbell JD, Pearson SD, Rind DM. Betibeglogene Autotemcel for Beta Thalassemia: Effectiveness and Value; Final Evidence Report. Institute for Clinical and Economic Review, July 19, 2022. https://icer.org/beta-thalassemia-2022/#timeline
(4)Beaudoin et al.