FDA Grants CAR T-Cell Therapy NXC-201 Orphan Drug Designation for Fatal Multiple Myeloma


During the phase 1b/2 trials, CAR T-cell therapy demonstrated significant and clinical survival benefits.

The FDA has granted NXC-201 (Nexcella), a next generation chimeric antigen receptor (CAR) T-cell therapy, orphan drug designation (ODD) for the treatment of multiple myeloma (MM). NXC-201 will receive 7 years of US market exclusivity, tax credits for clinical testing, and be waived from paying for the Prescription Drug User Fee Act (PDUFA), which is currently estimated at $3 million.1

Credit: LASZLO - stock.adobe.com

Credit: LASZLO - stock.adobe.com

“We are pleased to receive FDA’s ODD in MM for NXC-201, the only clinical-stage B-cell maturation antigen (BCMA)-targeted CAR-T cell therapy with no neurotoxicity observed in over 50 patients dosed to date,” said Ilya Rachman, MD, PhD, executive chairman of Nexcella, in a press release.

MM is a fatal blood cancer that stems from the excessive proliferation of plasma cells formed in bone marrow. Most patients have a high rate of relapse, and survival is poor among those who relapse following standard of care (protease inhibitors and immunomodulatory agents). Annually, more than 35,000 people are diagnosed with MM, and the market—$13.9 billion in 2017—is expected to hit $28.7 billion by 2027.

NXC-201 was initially evaluated in the phase 1b/2 NEXICART-1 (NCT04720313) trial, which included adults aged 18 years and older who had relapsed or refractory disease and had received a minimum of 3 prior lines of therapy (including a proteasome inhibitor or immunomodulatory agent). During phase 1 of NEXICART-1, investigators settled on a dose of 800 million CAR T-cells.2

In the ongoing phase 2 trial, NXC-201 is being evaluated for overall response rate (ORR), portion of patients with complete response (CR), and duration of response (DOR). Investigators are encouraged by the trial results; among 37 patients given 800 million CAR T-cells, 87% experienced partial response, 57% experienced CR (no cancer remains), and ORR was 92% compared to 75% who did not receive BCMA-targeted therapy.

While most US medical centers still do not offer CAR T-cell therapies like NXC-201 because of severe adverse events (AEs), this therapeutic alternative does not result in neurotoxicity, which is one of the most severe AEs associated with CAR T-cell therapy. The lack of a neurotoxicity AE has the potential to make NXC-201 a beneficial option for patients with relapsed or refractory disease.1,2

NXC-201 works by attaching itself to BCMA, a protein that lines the surface of myeloma cells, targeting and eliminating MM cancer cells while keeping healthy cells alive.2 The ODD granted to NXC-201 showcases that the product is intent for safe and effective treatment for the diagnosis or prevention of a rare disease/condition that impacts less than 200,000 people in the United States.1

“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,” said Gabriel Morris, president of Nexcella, in the press release.


  1. U.S. Food and Drug Administration Approves Orphan Drug Designation for NXC-201 as a Treatment for Multiple Myeloma. Nexcella, Inc. News Release. August 23, 2023. Accessed on August 23, 2023. globenewswire.com/news-release/2023/08/23/2730437/0/en/U-S-Food-and-Drug-Administration-Approves-Orphan-Drug-Designation-for-NXC-201-as-a-Treatment-for-Multiple-Myeloma.html
  2. Maia M. Positive early results seen for CAR T-cell therapy for myeloma in trial. Myeloma Research. News Release. May 1, 2023. Accessed on August 23, 2023. https://myelomaresearchnews.com/news/positive-early-results-seen-car-t-cell-therapy-myeloma-trial/
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