Akari Therapeutics Receives Positive Opinion on Orphan Drug Designation From EMA for Nomacopan for Treatment in Hematopoietic Stem Cell Transplantation
Akari Therapeutics, Plc recently announced the European Medicines Agency (EMA) has issued a positive opinion on the company’s application for orphan drug designation for nomacopan as a treatment in hematopoietic stem cell transplantation. The application included nomacopan clinical data from the treatment of patients with pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Akari is currently conducting a registrational Phase 3 clinical trial of nomacopan in pediatric HSCT-TMA.
“HSCT-TMA is serious, life-threatening condition with a mortality of 80% in severe forms, and yet there are no approved therapies for these patients,” said Rachelle Jacques, Akari President and CEO. “This positive opinion on nomacopan orphan drug designation from the EMA further validates the significant unmet need and, if granted, will support our urgent efforts to bring this potentially important treatment option forward to a regulatory filing.”
The positive opinion was issued by the Committee for Orphan Medicinal Products (COMP) of the EMA. Typically, the European Commission (EC) grants orphan drug designation 30 days after a positive opinion is issued. The EMA designates orphan drug status for medicines treating rare, life-threatening, or chronically debilitating diseases that meet certain criteria. If the designation is granted, Akari will receive scientific advice tailored for orphan drugs and, if a regulatory filing is approved, Akari also would receive extended market exclusivity.
The EMA orphan drug designation, if granted, will be an important addition to the Rare Pediatric Disease, Orphan Drug, and Fast Track designations already granted to nomacopan in pediatric HSCT-TMA by the U.S. Food and Drug Administration.
Akari Therapeutics, plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. Akari’s pipeline includes a Phase 3 clinical trial program investigating nomacopan for severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan for the treatment of pediatric HSCT-TMA. Akari’s pipeline also includes a clinical program developing nomacopan for adult HSCT-TMA and pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA). For more information, visit akaritx.com.
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