RMAT designation to obecabatagene autoleucel by FDA for patients with acute lymphoblastic leukaemia

May 2022 Pharma News Willem van Altena
CHRONIC MYLELOGENOUS LEUKEMIA (also called MYELOGENOUS LEUKEMIA), many MYELOBLASTS (Immature white blood cells or leukocytes), 250X. This is a human blood smear showing numerous Myeloblasts (immature white blood cells or leukocytes). It also shows an Eosinophil with reddish granules and a bilobed nucleus, and a BAND CELL (near the center, has a horseshoe-shaped nucleus) that develops into a NEUTROPHIL (the most common white blood cell). Large numbers of these is considered abnormal. These cells originate in the red bone marrow (MYELOID tissue), hence the name Myelogenous. Leukemia is a cancer of the blood forming tissues, and involves an abnormal proliferation of white blood, cells (leukocytes), many of which are immature, and do not perform normal functions. This great increase in leukocytes can also crowd out red blood cells and platelets. This image also shows numerous, smaller red blood cells (erythrocytes).

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.