OneChain Immunotherapeutics (OCI), member of CataloniaBio & HealthTech, has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA)to OC-1, a CAR T therapy against for the treatment of acute lymphoblastic leukemia (ALL), an aggressive subtype of T-cell leukemia. The company, which is part of the 1st generation of companies accelerated in the BCN Health Booster, has already started the CARxALL clinical trial to evaluate the safety and effectiveness of the product in humans. 

FDA Orphan Drug Designation, granted to drugs and biologics intended to treat, diagnose, or prevent diseases affecting fewer than 200,000 people in the United States, will provide the company with several benefits such as tax credits, exemption from certain regulatory fees, market exclusivity and mentoring during clinical development. 

“The Orphan Drug designation is a significant recognition that underscores the urgency and need for innovative treatments for this disease,” says Dr. Wilmar Castillo, director of Clinical Operations at OneChain Immunotherapeutics. “This is a crucial step in our journey to bring hope to patients who have no other therapeutic options,” she adds. 

Acute lymphoblastic leukemia is a type of hematological cancer characterized by the excessive production of immature lymphocytes, which invade the blood, bone marrow and lymphatic tissues, with the ability to spread to other parts of the body within a few months. This rare, aggressive cancer accounts for less than one-half of 1% of all cancers in the United States, and can be classified into several subtypes, each with even lower prevalence, depending on factors such as the type of lymphocyte affected and the stage of cell development. OCI’s therapy is focused on one of these subtypes. 

“The reality is that our product is directed at a very specific target, the CD1a antigen, which is expressed almost exclusively on the tumor cells of patients with cortical T-cell acute lymphoblastic leukemia,” explains Dr. Castillo. “For these patients, the first line of treatment is usually effective, but the prognosis for those who do not respond to existing therapies is very unfavorable.”