FDA Orphan Drug Designation (“ODD”) qualifies one-time treatment NXC-201 for 7 years of U.S. market exclusivity after approval
FDA ODD qualifies one-time treatment NXC-201 for Tax credits for qualified clinical testing. (Credit: HeungSoon from Pixabay)
Immix Biopharma, a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening blood disorder, Amyloid Light Chain (AL) Amyloidosis. 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 AL amyloidosis for NXC-201, the only clinical-stage CAR-T cell therapy in development for AL amyloidosis,” said Ilya Rachman, MD PhD, Immix Biopharma Chief Executive Officer, adding, “We are thrilled to potentially expand therapeutic options for relapsed and refractory AL amyloidosis patients, where we have observed to date in our NXC-201 clinical trials a 100% hematologic response rate and demonstrated organ responses in patient hearts, livers and kidneys, for AL amyloidosis patients who received a median of 6 earlier treatments that previously failed to halt the disease.”
Gabriel Morris, Immix Biopharma Chief Financial Officer, added, “We believe one-time treatment NXC-201 could offer AL amyloidosis patients a convenient therapeutic option.”
Source: Company Press Release