Nexcella announced that the FDA granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening form of blood cancer, multiple myeloma. NXC-201, a next generation CAR-T cell therapy, is currently being evaluated in a Phase 1b/2a clinical trial NEXICART-1 (NCT04720313).
The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. upon regulatory approval.
“We are pleased to receive FDA’s orphan drug designation in multiple myeloma for NXC-201, the only clinical-stage BCMA-targeted CAR-T cell therapy with no neurotoxicity observed in over 50 patients dosed to date,” said Ilya Rachman, MD PhD, Executive Chairman of Nexcella. Dr. Rachman continued, “We are thrilled to potentially expand therapeutic options for multiple myeloma patients, while eliminating the most feared adverse effect of this therapeutic class, neurotoxicity.”
Gabriel Morris, President of Nexcella, added, “Orphan drug designation for NXC-201 represents a substantial value creating step along our path to unlocking planned wide adoption of CAR-T technology by transitioning it to an outpatient domain.”
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