Akari Therapeutcis (AKTX) Receives FDA Orphan Drug Designation for Coversin as PNH Treatment

September 12, 2016 7:22 AM EDT
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Akari Therapeutics (Nasdaq: AKTX) announced that the U.S. Food and Drug Administration (FDA) has granted to Akari an Orphan Drug Designation for recombinant protein inhibitor of complement factor 5 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). Coversin, Akari’s lead clinical product, which falls within the Orphan Drug Designation, is a second-generation complement inhibitor that acts on complement component-C5, preventing release of C5a and formation of C5b-9 (also known as the membrane attack complex or MAC).

PNH is an ultra-rare, life-threatening and debilitating disease of the blood with an estimated 8,000-10,000 patients across North America and Europe. Due to an acquired genetic deficiency, uncontrolled complement activation in PNH patients allows their own complement system to attack and destroy blood cells, leading to life-threatening complications.

"We are pleased to receive Orphan Drug Designation in the United States for PNH, and now have this designation for PNH and Guillain Barre Syndrome, or GBS, in the US and EU," said Dr. Gur Roshwalb, CEO of Akari. "We have continued to see complete complement inhibition and symptom control in a PNH patient with resistance to eculizumab, who has been self-administering subcutaneous Coversin for over 7 months. We believe that Coversin, when approved, could provide important benefits for all patients with PNH.”

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at the time of designation. Orphan Drug Designation provides the sponsor certain benefits and incentives, including a period of marketing exclusivity if regulatory approval of the drug is ultimately received for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants, and the waiver of certain administrative fees. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be received.

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