FDA Grants Orphan Drug Designation to Felzartamab for Kidney Transplant Recipients


Previously, felzartamab received orphan drug designation and breakthrough therapy designation for the treatment of primary membranous nephropathy.

The FDA has granted Orphan Drug Designation to felzartamab (Human Immunology Biosciences) for the treatment of antibody-mediated rejection in individuals who received a kidney transplant, according to a press release. Felzartamab is an investigational therapeutic human monoclonal antibody that is directed against CD38 and selectively depletes the CD38+ plasma cells, improving clinical outcomes in a range of diseases that include pathogenic antibodies.1

esthetic handdrawn highlighted illustration of human kidney. | Image Credit: mi_viri - stock.adobe.com

Image Credit: mi_viri - stock.adobe.com

"Following the FDA granting of breakthrough therapy designation for felzartamab in primary membranous nephropathy (PMN), we are encouraged to receive orphan drug designation for felzartamab for antibody-mediated rejection," Uptal Patel, MD, chief medical officer at HI-Bio, said in the press release. "Along with our academic collaborators, we look forward to submitting clinical data from the ongoing study of felzartamab in antibody-mediated rejection to a medical conference this year. We are confident in the clinical progress of our anti-CD38 cellular depletion strategy, which to date, has resulted in proof-of-concept data in multiple severe immune-mediated diseases including [AMR], IgA nephropathy and [PMN]."1

Previously in May 2023, felzartamab received orphan drug designation for the treatment of PMN, which was based on data from the phase 2 studies M-PLACE and NewPLACE. M-PLACE was a phase 1b/2a proof-of-concept study that assessed the safety and efficacy of the drug in adults with aPLA2R-positive PMN in a 9-dose course spanning across 5 months. Investigators enrolled 31 patients in this first study and data were presented as an oral presentation at the American Society of Nephrology Kidney Week 2023 Annual Meeting.2-4

In the NewPLACE phase 2 study, investigators enrolled 24 patients to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of 2- and 5-dose courses over 2 weeks and 2 months, respectively. They also evaluated retreatment with felzartamab in individuals with a PLA2R-positive PMN for immunosuppressive therapy, according to the press release.3

Key Takeaways

  1. The FDA granted Orphan Drug Designation for felzartamab, an investigational drug, for treating antibody-mediated rejection (AMR) in kidney transplant recipients.
  2. elzartamab is being studied in other immune-mediated diseases including IgA nephropathy, primary membranous nephropathy (PMN), and lupus nephritis. It has shown promising results in PMN trials.
  3. Felzartamab also has Breakthrough Therapy Designation for PMN, aiming to speed up development and approval for this serious condition.

Based on the M-PLACE study, the FDA also granted breakthrough designation to felzartamab for PMN, according to another press release.4

"The FDA's decision to grant felzartamab breakthrough therapy designation is recognition of the promising data we have collected to date, as well as an acknowledgement of the need for major advances over available therapies in the treatment of patients with PMN,” Patel said in another press release. "We believe that the cellular depletion strategy with felzartamab in PMN is applicable to many more immune-mediated diseases driven by antibodies produced in CD38+ plasma cells. For that reason, we are currently developing felzartamab in multiple diseases including PMN, IgA nephropathy, [AMR], and lupus nephritis."4

Orphan drug designation is assigned to the development of drugs and biologics that are intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States, and breakthrough designation is designed to expedite the development and review of drugs intended to treat a serious or life-threatening condition where preliminary clinical evidence shows that the drug could provide substantial improvements to current available therapy.1,4

  1. HI-Bio Receives FDA Orphan Drug Designation for Felzartamab for the Treatment of Antibody-Mediated Rejection (AMR) in Kidney Transplant Recipients. News release. HI-Bio. March 21, 2024. Accessed March 25, 2024. https://prnmedia.prnewswire.com/news-releases/hi-bio-receives-fda-orphan-drug-designation-for-felzartamab-for-the-treatment-of-antibody-mediated-rejection-amr-in-kidney-transplant-recipients-302095317.html
  2. HI-Bio Receives FDA Orphan Drug Designation for Felzartamab for the Treatment of Membranous Nephropathy. News release. HI-Bio. May 25, 2023. Accessed March 25, 2024. https://prnmedia.prnewswire.com/news-releases/hi-bio-receives-fda-orphan-drug-designation-for-felzartamab-for-the-treatment-of-membranous-nephropathy-301834111.html
  3. HI-Bio Announces Positive Phase 2 Data on Felzartamab for the Treatment of Primary Membranous Nephropathy. News release. HI-Bio. April 11, 2023. Accessed March 25, 2024. https://hibio.com/news/hi-bio-announces-positive-phase-2-data-on-felzartamab-for-the-treatment-of-primary-membranous-nephropathy
  4. HI-Bio Announces Felzartamab Granted Breakthrough Therapy Designation by U.S. Food and Drug Administration for Primary Membranous Nephropathy (PMN). News release. HI-Bio. October 31, 2023. Accessed March 25, 2024. https://prnmedia.prnewswire.com/news-releases/hi-bio-announces-felzartamab-granted-breakthrough-therapy-designation-by-us-food-and-drug-administration-for-primary-membranous-nephropathy-pmn-301973164.html
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