The drug is currently being assessed in a phase 1b/2a clinical trial.
Nexcella has announced that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) to NXC-201 for the treatment of multiple myeloma. The drug is currently being assessed in the phase 1b/2a NEXICART-1 clinical trial.
The ODD qualifies the drug for seven years of US market exclusivity once it is approved, tax credits for qualified clinical testing and a waiver of the Prescription Drug User Fee (currently nearing $3m for a new drug). The FDA grants this status to drugs and biologics intended for safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the US.
Ilya Rachman, executive chairman of Nexcella, commented: “We are pleased to receive FDA’s ODD 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.”
Gabriel Morris, president of Nexcella, added: “ODD 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.”