Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment
Retrieved on:
Monday, February 12, 2024
FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
Key Points:
- FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
- The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV).
- CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
- FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition.