With ODD, NXC-201 is now eligible for seven years of US market exclusivity in the US upon regulatory approval and will also get tax credits for qualified clinical testing and a waiver of the Prescription Drug User Fee
A histopathological image of multiple myoloma. (Credit: KGH from Wikimedia Commons)
Immix Biopharma has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its next-generation CAR-T cell therapy, NXC-201. to treat life-threatening blood cancer and multiple myeloma.
NXC-201 is being developed by ImmixBio’s subsidiary Nexcellaz and is currently being assessed in a Phase 1b/2a NEXICART-1 clinical trial.
FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are designed for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that impact fewer than 200,000 persons in the US.
With ODD, NXC-201 is now eligible for seven years of US market exclusivity in the US upon regulatory approval.
It will also get tax credits for qualified clinical testing and a waiver of the Prescription Drug User Fee.
Immix Biopharma CEO Ilya Rachman said: “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.
“We are thrilled to potentially expand therapeutic options for multiple myeloma patients, while eliminating the most feared adverse effect of this therapeutic class, neurotoxicity.”
NXC-201 is being developed for relapsed/refractory multiple myeloma and relapsed/refractory AL Amyloidosis.
In the Phase 1b/2a NEXICART-1 study, the initial response rates were observed at 92% and 100% in patients with multiple myeloma and AL amyloidosis, respectively.
The therapy has the potential to be the world’s first out-patient CAR-T, Immix Biopharma said.
Additionally, the company has IMX-110, a Tissue-Specific Therapeutic (TSTx) asset, in Phase 1b/2a trials as a monotherapy and IMMINENT-01 combination trial with BeiGene’s anti-PD-1 antibody tislelizumab.
Nexcella president Gabriel Morris said: “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.”