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The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics 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. The FDA has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening form of blood cancer, multiple myeloma.
Clinical-stage Biopharmaceutical company Nexcella announced that the FDA has granted an ODD for its next-generation CAR-T cell therapy, NXC-201, which is currently being evaluated in a Phase Ib/IIa clinical trial NEXICART-1 (NCT04720313).
The ODD provides certain benefits which include financial incentives to support clinical development and the potential for up to seven years of marketing exclusively 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,” commented Ilya Rachman, MD PhD, Executive Chairman of Nexcella.
Most patients, despite initial remission, are likely to relapse. There are 35,730 patients in the United States diagnosed with multiple myeloma each year.
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Nexcella’s lead product candidate, next-generation BCMA-target CAR-T NXC-201 for relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis has produced 92% and 100% response rates in each indication, respectively, as of February 9, 2023, across 58 patients.
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.”
Given the ODD, NXC-201 qualifies for seven years of U.S. market exclusivity after approval, a tax credit for qualified clinical testing and a Waiver of the Prescription Drug User Fee.
“We are thrilled to potentially expand therapeutic options for multiple myeloma patients while eliminating the most feared adverse effect of this therapeutic class, neurotoxicity,” relayed Dr. Rachman.
Source: Nexcella Press Release
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