FDA Grants Orphan Drug Status to Anti-Properdin Antibody for PNH

The Food and Drug Administration (FDA) has granted Orphan Drug designation to NM5072 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

NM5072 is an investigational monoclonal antibody that selectively blocks properdin, a protein of the complement alternative pathway. Inhibition of properdin is expected to prevent lysis of red blood cells and thus improve anemia in patients with PNH.

In a phase 1 trial, 48 healthy volunteers were administered multiple doses of NM5072. Results showed the anti-properdin antibody was well tolerated and safe. Additionally, the duration of total alternative pathway inhibition was observed to be dose dependent. 

“This Orphan Drug designation approval represents a remarkable stride in therapeutic innovation, showcasing NM5072’s distinct capability to selectively inhibit the alternative pathway while safeguarding the classical pathway essential for combating infections in PNH patients,” said Robert Bard, VP Regulatory Affairs, NovelMed Therapeutics.

The Company is planning on a phase 2 trial that will include treatment-naïve PNH patients.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.