Biogen Gets FDA Breakthrough Therapy Status for Felzartamab to Treat Kidney Transplant Rejection

Biogen Inc., a renowned biotechnology firm, has announced that its investigational drug felzartamab has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA). This designation is for the treatment of late antibody-mediated rejection (AMR) in kidney transplant patients without T-cell mediated rejection. The FDA awards BTD to therapies that have shown potential to offer significant improvements over existing treatments for serious or life-threatening conditions.

Felzartamab is an investigational anti-CD38 monoclonal antibody. The drug has demonstrated clinical proof of concept and is considered a potential first-in-class therapeutic candidate for various rare immune-mediated conditions. Planning is underway for Phase 3 development of the drug. The BTD status provides an opportunity for more intensive interaction with the FDA and benefits from Fast Track designation features, which could expedite the review and approval process.

The data that supported the BTD for felzartamab were published in the New England Journal of Medicine and also presented at the European Renal Association Congress in Stockholm in May 2024. According to Travis Murdoch, Head of Human Immunology Biosciences at Biogen, antibody-mediated rejection is a leading cause of kidney transplant failure, and there is a significant unmet medical need for effective treatments. The breakthrough therapy designation will enable Biogen to work closely with the FDA to accelerate the development of felzartamab for AMR.

Felzartamab has previously received both Breakthrough Therapy Designation and Orphan Drug Designation (ODD) from the FDA for the treatment of primary membranous nephropathy (PMN) and ODD for the treatment of AMR in kidney transplant patients. Phase 2 studies for AMR, PMN, and IgA nephropathy (IgAN) have been completed, and Biogen plans to initiate Phase 3 trials for these conditions in 2025.

Biogen acquired Human Immunology Biosciences in July 2024. Felzartamab, which was originally developed by MorphoSys AG for the treatment of multiple myeloma, is a human monoclonal antibody targeting CD38, a protein found on mature plasma cells. Clinical studies have shown that felzartamab can selectively deplete CD38+ plasma cells, which could potentially improve clinical outcomes in various diseases driven by pathogenic antibodies. Human Immunology Biosciences holds exclusive rights to develop and commercialize felzartamab globally, excluding China, including Macau and Hong Kong and Taiwan.

Antibody-mediated rejection (AMR) is a leading cause of kidney transplant failure in the U.S., affecting approximately 12% of kidney transplant recipients annually. AMR has become the primary cause of late graft loss among kidney transplant patients, and effective treatment options for chronic AMR remain limited.

Founded in 1978, Biogen is a pioneering biotechnology company dedicated to advancing innovative science to develop new medicines that transform patients' lives. The company leverages a deep understanding of human biology and different modalities to create first-in-class treatments that deliver superior outcomes. Employing a strategy of taking bold risks balanced with a focus on return on investment, Biogen aims to deliver long-term growth and value for its shareholders and communities.

In summary, Biogen’s felzartamab has received FDA Breakthrough Therapy Designation for the treatment of AMR in kidney transplant patients, highlighting its potential to address a significant unmet medical need. Biogen remains committed to advancing felzartamab through clinical development, with plans for Phase 3 trials in several conditions slated to begin in 2025.