Nomacopan receives Orphan Drug Designation for Hematopoietic Stem Cell Transplantation

European Commission Grants Orphan Drug Designation to Nomacopan: A Potential Breakthrough in Hematopoietic Stem Cell Transplantation Treatment

Akari Therapeutics, a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, announced that the European Commission granted orphan drug designation to nomacopan as a treatment in hematopoietic stem cell transplantation following the European Medicines Agency positive opinion in June. Akari is currently conducting a registrational Phase 3 study of nomacopan in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Additionally, the company is moving forward into a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA with enrollment expected to begin in 2024.

This orphan drug designation is an important addition to the Rare Pediatric Disease (which entitles Akari to a Priority Review Voucher at approval), Orphan Drug (pediatric and adult), and Fast Track (pediatric) designations already granted to nomacopan in HSCT-TMA by the U.S. Food and Drug Administration.

Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. There are currently no approved therapies for HSCT-TMA, a rare condition with an 80% mortality rate in high-risk (severe) pediatric and adult patients.

Published on : 28th July, 2023