NEW YORK and LONDON, Aug. 30, 2019 (GLOBE NEWSWIRE) — Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement (C5) and/or leukotriene (LTB4) systems are implicated, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for nomacopan for the treatment of hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA).
Orphan drug designation for nomacopan follows Fast Track designation that the Company received from the FDA earlier in August 2019 for the same indication in pediatric patients. The Company continues to progress towards a pivotal trial for HSCT-TMA with nomacopan, which is expected to start in the fourth quarter of 2019.
“We are pleased to obtain orphan drug designation for nomacopan in HSCT-TMA, a devastating rare disease for which there are currently no approved treatments,” said Clive Richardson, Chief Executive Officer of Akari Therapeutics. “The granting of orphan drug designation and Fast Track designation by the FDA for nomacopan underscores the significant unmet medical need in this disease. We look forward to taking advantage of the opportunities that FDA orphan drug designation and Fast Track designation provide across all stages of drug development in order to bring this potential new treatment option to patients as rapidly as possible.”
Orphan drug designation by the FDA is granted to promote the development of drugs that target conditions affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies Akari for various benefits, including seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.
About Akari Therapeutics
Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari’s lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity. Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in both AKC and BP.