hal-gatewood-_jbclosdsd4-unsplash

FDA grants ODD to NXC-201 as multiple myeloma treatment

Betsy Goodfellow | August 24, 2023 | News story | Medical Communications FDA, Nexcella, ODD, Oncology, multiple myeloma 

Nexcella has announced that the US Food and Drug Administration (FDA) has granted 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.

This 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, MD PhD, executive chairman of Nexcella, commented: “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.”

Advertisement

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.”

Betsy Goodfellow

Related Content

Rethinking oncology trial endpoints with generalised pairwise comparisons

For decades, oncology trials have been anchored to a familiar set of endpoints. Overall survival …

brain_anatomy_medical_head_skull_digital_3_d_x_ray_xray_psychedelic_3720x2631_1

Alto Neuroscience’s schizophrenia treatment granted FDA Fast Track designation

Alto Neuroscience has announced that its investigational treatment for cognitive impairment associated with schizophrenia (CIAS) …

FDA approves Moderna’s updated COVID-19 vaccines targeting new variant

FDA approves Moderna’s updated COVID-19 vaccines targeting new variant

The US Food and Drug Administration (FDA) has approved Moderna’s updated COVID-19 vaccines, Spikevax and …

The Gateway to Local Adoption Series

Latest content