FDA Grants Rare Pediatric Disease Designation to Akari’s Experimental Therapy for Pediatric HSCT-TMA

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to Akari Therapeutics’ nomacopan for the treatment of pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).

Thrombotic microangiopathy (TMA) is a common complication occurring post-hematopoietic stem cell transplantation (HSCT) that can be fatal. It causes microvascular thrombosis, leading to thrombocytopenia, ischemic tissue damage, and microangiopathic hemolytic anemia.

Akari’s lead asset, nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 activity, which is currently being investigating in a phase 3 clinical trial for severe HSCT-TMA. Akari has already received Orphan Drug and Fast Track designations from the FDA for nomacopan for the treatment of 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 at Akari Therapeutics. “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.”

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. Most recently, Marinus Pharmaceuticals sold it priority review voucher to Novo Nordisk for $110 million.

Photo: Rachelle Jacques, president and CEO at Akari Therapeutics