FDA grants orphan drug designation to off-the-shelf CAR-T for advanced multiple myeloma
Click Here to Manage Email Alerts
The FDA granted orphan drug designation to ALLO-605, a chimeric antigen receptor T-cell therapy for adults with relapsed or refractory multiple myeloma.
ALLO-605 (Allogene Therapeutics) is an allogeneic, gene-edited CAR T-cell therapy that targets the B-cell maturation antigen (BCMA) on the surface of cancer cells. The investigational agent was previously given fast track designation by the FDA.
The off-the-shelf CAR T-cell therapy is derived from healthy donor-induced pluripotent stem cells and was developed using Allogene’s proprietary TurboCAR platform to improve their potency and function.
“Orphan-drug designation marks an important step [toward] developing our anti-BCMA portfolio for patients with multiple myeloma and making allogeneic [CAR-T] products readily available for patients,” Rafael Amado, MD, executive vice president of research and development and chief medical officer of Allogene, said in a company-issued press release. “We look forward to providing an update on our BCMA clinical assets by the end of the year, with an eye toward prioritizing a strategy for the next stage of development.”
ALLO-605 is currently being evaluated as part of the phase 1 IGNITE trial. The dose-escalation study aims to evaluate the safety, feasibility and recommended phase 2 dose of ALLO-605 for patients with relapsed or refractory multiple myeloma or other BCMA-positive malignancies. The trial uses the CAR T-cell therapy in combination with ALLO-647 (Allogene Therapeutics) — a CD52-directed monoclonal antibody — as part of the study’s lymphodepletion regimen prior to ALLO-605 infusion.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Read more about
Click Here to Manage Email Alerts