FDA grants orphan drug status to HI-Bio’s felzartamab for AMR

Clinical trials demonstrated felzartamab’s potential to improve outcomes in diseases caused by pathogenic antibodies.

Vishnu Priyan March 22 2024

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Human Immunology Biosciences’ (HI-Bio) investigational therapeutic felzartamab, to treat antibody-mediated rejection (AMR) in kidney transplant recipients.

The designation is a significant step in the development of felzartamab, a monoclonal antibody targeting cluster of differentiation 38 (CD38), which is involved in the production of pathogenic antibodies. It selectively depletes CD38+ plasma cells.

Clinical trials demonstrated its potential to improve outcomes in diseases caused by these pathogenic antibodies.

HI-Bio is developing felzartamab for immune-mediated diseases including AMR, IgA nephropathy, lupus nephritis and primary membranous nephropathy (PMN).

The FDA has previously awarded felzartamab both breakthrough therapy and orphan drug statuses for PMN treatment.

HI-Bio acquired exclusive global rights to felzartamab, excluding Greater China, through a licensing agreement with MorphoSys in 2022.

The FDA’s ODD provides HI-Bio with development incentives such as tax credits for qualified clinical studies, a waiver of FDA application fees and the potential for seven-year market exclusivity upon regulatory approval.

AMR is a leading cause of kidney transplant failure with no current effective treatment.

The condition is associated with donor-specific antibody production by plasma cells and the infiltration of natural killer cells, which contribute to microvascular inflammation.

HI-Bio chief medical officer Uptal Patel said: “Following the FDA’s granting of breakthrough therapy designation for felzartamab in primary membranous nephropathy, we are encouraged to receive orphan drug designation for felzartamab for antibody-mediated rejection.

“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 antibody-mediated rejection, IgA nephropathy and primary membranous nephropathy.” 

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