The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) status to gene therapy candidate obecabatagene autoleucel (obe-cel) for treating patients with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL).
The obe-cel is an autologous CAR-T cell therapy developed by Autolus Therapeutics plc, a biopharmaceutical company developing next-generation programmed T cell therapies. Its lead gene therapy candidate has received RMAT designation by the FDA. The FDA grants the RMAT designation to candidate treatment options that have the potential to address significant unmet needs for patients with serious/life-threatening diseases.
“RMAT designation is an important regulatory milestone for obe-cel and highlights its potential to address the unmet medical need for adult patients with relapsed and refractory B-ALL,” said Dr Dristian Itin, Chief Executive Officer of Autolus. “RMAT designation from FDA, PRIME designation from EMA, and ILAP designation from MHRA facilitate regulatory interactions with key health authorities and supports our drive to bring this innovative therapy to patients as quickly as possible.”
The efficacy and safety of obe-cel for B-cell ALL patients will be evaluated in the phase-2 FELIX clinical trial (NCT04404660). This multicentre, international study intends to enrol around 140 patients across the USA and Europe. The study’s primary endpoint is overall response rate, and the secondary endpoints include duration of response, MRD negative CR rate and safety.