FDA grants ODD to Nexcella’s NXC-201 as treatment for amyloid light chain amyloidosis
Nexcella has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to NXC-201 for the treatment of blood disorder, amyloid light chain (AL) amyloidosis.
NXC-201 is a next generation CAR T-cell therapy, and is currently being assessed in the phase 1b/2a NEXICART-1 clinical trial.
This ODD qualifies NXC-201 for seven years of market exclusivity following its approval, tax credits for qualified clinical testing and a waiver of the Prescription Drug User Fee (currently nearly $3m for a new drug). The FDA’s Office of Orphan Products Development grants this status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the US.
Ilya Rachman MD PhD, executive chairman of Nexcella, commented: “We are pleased to receive FDA’s ODD in AL amyloidosis for NXC-201, the only clinical-stage CAR T-cell therapy in development for AL amyloidosis. 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% haematologic 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, president of Nexcella, added: “We believe one-time treatment NXC-201 could offer AL amyloidosis patients a convenient therapeutic option.”
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