US FDA Approves Orphan Drug Designation for NXC-201 as a Treatment for Multiple Myeloma
Nexcella, Inc. recently announced the US FDA has 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 US. 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 US 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. “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.”
Multiple myeloma (MM) is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 35,730 patients in the United States diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, remains poor. The $13.9 billion Multiple Myeloma market in 2017 is expected to reach $28.7 billion in 2027 according to Wilcock, et al. Nature Reviews.
Nexcella, Inc. is a Los Angeles, CA-based clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our lead candidate, next generation BCMA-targeted 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. We believe NXC-201 has potential to be the world’s first outpatient CAR-T. Our N-GENIUS platform allows us to discover, develop, and manufacture cutting-edge cell therapies for patients in need. For more information, visit www.nexcella.com.
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