Pharma Focus America

FDA Approves Orphan Drug Designation for NXC-201 as Treatment for Multiple Myeloma

Thursday, August 24, 2023

Nexcella, Inc., a clinical-stage biopharmaceutical company specializing in the development of novel cell therapies for various medical indications, has announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to NXC-201 for treating multiple myeloma, a severe form of blood cancer. NXC-201, an advanced CAR-T cell therapy, is currently undergoing assessment in a Phase 1b/2a clinical trial known as NEXICART-1 (NCT04720313).

Orphan Drug Designation is provided by the FDA's Office of Orphan Products Development to drugs and biologics intended to effectively and safely address rare diseases or conditions affecting fewer than 200,000 individuals in the United States. This designation comes with several benefits, including financial incentives to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. after regulatory approval.

Dr. Ilya Rachman, MD PhD, Executive Chairman of Nexcella, expressed satisfaction with the orphan drug designation for NXC-201, highlighting its distinction as a clinical-stage BCMA-targeted CAR-T cell therapy that has demonstrated no instances of neurotoxicity in over 50 treated patients. Dr. Rachman emphasized the potential to expand treatment options for multiple myeloma patients while eliminating the neurotoxicity concern associated with this therapeutic class.

Gabriel Morris, President of Nexcella, pointed out that the orphan drug designation for NXC-201 signifies a significant step towards making CAR-T technology more accessible by transitioning it to an outpatient setting.

Multiple myeloma is an incurable blood cancer originating in the bone marrow's plasma cells, characterized by their excessive proliferation. Despite initial remission, a majority of patients are prone to relapse. Around 35,730 patients are diagnosed with multiple myeloma each year in the United States. Patients who do not respond to standard treatments face a grim prognosis. The multiple myeloma market was valued at $13.9 billion in 2017 and is projected to reach $28.7 billion by 2027 according to Wilcock et al. in Nature Reviews.

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