Pharma Focus America

FDA Grants Orphan Drug Designation for Nexcella NXC-201 in the Treatment of Amyloid Light Chain (AL) Amyloidosis

Friday, September 22, 2023

Nexcella, Inc. ("Nexcella"), a clinical-stage biopharmaceutical company specializing in the development of innovative cell therapies for oncology and other medical conditions, has announced that it received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for NXC-201 in the treatment of Amyloid Light Chain (AL) Amyloidosis, a severe blood disorder. NXC-201, a next-generation CAR-T cell therapy, is currently undergoing evaluation in the Phase 1b/2a clinical trial NEXICART-1 (NCT04720313).

Orphan Drug Designation is granted by the FDA's Office of Orphan Products Development to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that typically affect fewer than 200,000 people in the U.S. This designation provides several benefits, including financial incentives and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

Dr. Ilya Rachman, MD PhD, Executive Chairman of Nexcella, expressed satisfaction with the FDA's orphan drug designation for NXC-201 in AL amyloidosis, emphasizing that NXC-201 is the only clinical-stage CAR-T cell therapy in development for this condition. He also highlighted promising results observed in NXC-201 clinical trials, such as a 100% hematologic response rate and positive organ responses in patient hearts, livers, and kidneys. These results are particularly encouraging for AL amyloidosis patients who have previously undergone a median of six unsuccessful treatments in their fight against the disease. Gabriel Morris, President of Nexcella, added that they believe one-time treatment with NXC-201 could offer a convenient therapeutic solution for AL amyloidosis patients.

AL amyloidosis is a rare systemic disorder resulting from abnormalities in plasma cells found in the bone marrow. Misfolded amyloid proteins produced by these cells accumulate in and around tissues, nerves, and organs, progressively impairing their function. This leads to progressive and widespread organ damage and is associated with high mortality rates.

AL amyloidosis affects approximately 30,000 to 40,000 patients in the U.S. and Europe, with an estimated 3,000 to 4,000 new cases diagnosed annually in the U.S. The estimated global annual incidence is around 15,000 patients.

According to Grand View Research, the Amyloidosis market was valued at $3.6 billion in 2017, with expectations to reach $6 billion by 2025.

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