NXC-201 Gains FDA Orphan Drug Designation for AL Amyloidosis


NXC-201, a next-generation chimeric antigen receptor T-cell therapy, has potential to expand as treatment for patients with amyloid light chain amyloidosis.

  • NXC-201 is the only clinical-stage chimeric antigen receptor (CAR) T-cell therapy in development for amyloid light chain (AL) amyloidosis.

  • With an orphan drug designation (ODD) from the FDA, NXC-201 qualifies for up to 7 years of market exclusivity upon regulatory approval.

  • NXC-201 is also being evaluated in the phase 1b/2a NEXICART-1 study (NCT04720313) for the treatment of patients with multiple myeloma.

The FDA has granted an ODD for NXC-201 (formerly HBI0101) for the treatment of AL amyloidosis.1

With this ODD, one-time treatment with NXC-201 qualifies for 7 years of United States market exclusivity after approval, tax credits for qualified clinical testing, and waiver of the prescription drug user fee, upon regulatory approval.

“We are pleased to receive FDA’s orphan drug designation in AL amyloidosis for NXC-201, the only clinical-stage CAR-T cell therapy in development for AL amyloidosis,” said Ilya Rachman, MD, PhD, executive chairman of Nexcella, in a press release. “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% hematologic 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.”

Amyloidosis © ChrWeiss - stock.adobe.com

Amyloidosis © ChrWeiss - stock.adobe.com

NXC-201, a next generation CAR-T cell therapy. The development of the product aims to offer a different mechanism than FDA-approved CAR T cell agents, including tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta).2

The product is currently being evaluated in a phase 1b/2a trial titled NEXICART-1. In the dose-escalation phase of the trial (part A), patients will be treated with doses of CAR-positive T cells at 150 × 106, 450 × 106, 800 × 106, or 1200 × 106. In part B, the expansion phase of the study, patients will be treated at a dose between 450 × 106 to 800 × 106 CAR-positive T cells.3

Patients aged 18 years and older are eligible for enrollment in the study if they have a diagnosis of relapsed or refractory multiple myeloma, have received at least 3 prior lines of therapy, including with a proteasome inhibitor, immunomodulatory therapy, and at least 1 antibody therapy, and an ECOG performance status 0-2. Patients must have measurable disease and recover to ≤ grade 2 or baseline of any non-hematologic toxicities resulting from prior treatments, excluding alopecia and grade 3 neuropathy. Additionally, women of child-bearing potential must agree to use effective contraception throughout the duration of the trial and have a negative serum pregnancy test prior to treatment.

The primary end point being assessed in part A of the trial is the determination of a maximum tolerated dose and the confirmation of the selected dose tested in part B. Secondary end points include overall survival and progression-free survival.

“We believe one-time treatment NXC-201 could offer AL amyloidosis patients a convenient therapeutic option,” said Gabriel Morris, president of Nexcella, in a press release.1

  1. U.S. Food and Drug Administration approves orphan drug designation for Nexcella NXC-201 as a treatment for amyloid light chain (AL) amyloidosis. News release. Nexcella, Inc. September 21, 2023. Accessed September 21, 2023. https://tinyurl.com/3kkaf4ww
  2. Pipeline. Nexcella. Accessed September 21, 2023. https://tinyurl.com/43cjk944
  3. NXC-201 (Formerly HBI0101) Multiple Myeloma. ClinicalTrials.gov. Updated May 3, 2023. Accessed September 21, 2023. https://classic.clinicaltrials.gov/ct2/show/NCT04720313