Akari Therapeutics Granted FDA Rare Pediatric Disease Designation of Nomacopan for the Treatment of Pediatric HSCT-TMA

Akari Therapeutics, Plc recently announced the US FDA has granted the Rare Pediatric Disease Designation to nomacopan for the treatment of pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). The Rare Pediatric Disease Designation is an important addition to the Orphan Drug and Fast Track designations previously granted by the FDA for nomacopan in pediatric HSCT-TMA.

“Families with children who have a hematopoietic stem cell transplant-related TMA face grim prospects with high mortality rates and no approved treatment options,” said Rachelle Jacques, President and CEO of Akari. “The Rare Pediatric Disease Designation was created by the FDA because families need so much more than hope for their children, they need meaningful treatment options. We are pleased the FDA has recognized the significant needs that exist for children with HSCT-TMA, and we will continue to urgently pursue our mission to advance nomacopan to approval on their behalf.”

The FDA Rare Pediatric Disease Designation and Voucher Program is a recognition of the significant need that exists for approved treatments in rare pediatric diseases and is intended to encourage development of these treatments. Under this program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a Priority Review Voucher (PRV). A PRV is valuable because it can be redeemed to obtain priority review for a subsequent marketing application for a different product or may be sold to a third party.

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 pre-clinical research of long-acting PAS-nomacopan in geographic atrophy (GA). For more information, visit akaritx.com.