PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Ruxoprubart Scores FDA Orphan Drug Designation for PNH Treatment
NovelMed Therapeutics 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.
Ruxoprubart is currently undergoing a Phase II trial in treatment-naïve PNH patients, which aims to evaluate the safety and efficacy of the drug and bringing us one step closer to providing an effective and innovative therapy for PNH patients in USA and around the world.
“We are delighted by the FDA’s decision to grant orphan drug designation to Ruxoprubart, underscoring the pressing need for innovative therapeutic solutions for patients with PNH,” says Robert Bard, VP Regulatory Affairs. This ODD approval marks a significant advancement in the therapeutic landscape with Ruxoprubart ‘s unique ability to selectively block the alternative pathway (AP) while preserving the classical pathway required for clearing infections in PNH patients.
The ODD from the US FDA is a special designation granted to facilitate the development and assessment of prospective new medications. The FDA’s Office of Orphan Products Development bestows orphan status upon drugs in development for the treatment, diagnosis, or prevention of rare diseases or conditions impacting fewer than 200,000 people in the US.
