Home / FDA News / FDA Grants Fast Track Designation to Akari’s Nomacopan for Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy

FDA Grants Fast Track Designation to Akari’s Nomacopan for Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy

NEW YORK and LONDON, Aug. 14, 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 and/or leukotriene systems are implicated, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for nomacopan for the treatment of hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) in pediatric patients. The Fast Track designation supports Akari’s plans for expedited approval of nomacopan for HSCT-TMA, with the pivotal trial expected to commence in the fourth quarter of 2019.

Fast Track designation is intended to facilitate the development and expedite the review of new drugs which show promise in treating serious or life-threatening conditions and address unmet medical needs. Drugs that receive this designation benefit from more frequent communications and meetings with FDA to review the drug’s development plan including the design of the proposed clinical trials, use of biomarkers and the extent of data needed for approval. Drugs with Fast Track designation may qualify for priority review to expedite the FDA review process, if relevant criteria are met.

HSCT-TMA is an orphan condition with an estimated fatality rate of more than 80% in children with the severe diseaseThere are currently no approved treatments for HSCT-TMA but there is strong evidence for the role of complement and potentially LTB4 in the etiology of the disease.

In September 2018, Akari announced that in the first two HSCT-TMA patients treated with nomacopan as part of a UK named patient program, it had observed disease resolution by way of a rapid reduction of the markers of complement activation as well as normalization of markers that are elevated in HSCT-TMA patients (platelet count, red blood cell fragments, thrombocytopenia, elevated LDH and hypertension). On the basis of existing treatment data from these HSCT-TMA patients and a further 20 cumulative patient-years of data from other patients treated with nomacopan, Akari is working with the FDA Model Informed Drug Development (MIDD) Program to optimize the pediatric dosing with nomacopan for the planned pivotal HSCT-TMA trial.

Clive Richardson, interim CEO of Akari Therapeutics, said, “Fast Track designation provides our HSCT-TMA clinical program a pathway for an expedited approval for nomacopan, and we plan to commence a pivotal trial in the fourth quarter of 2019. We see HSCT-TMA as a gateway into a range of other poorly treated orphan TMAs in both pediatric and adult patients, and are optimistic about the potential for nomacopan to offer an improved standard of care for these orphan conditions with high mortality rates.”

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 AKC, BP and other orphan inflammatory conditions. Akari is also developing other tick derived proteins, including longer acting versions.

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