Akari Therapeutics (NASDAQ:AKTX) received FDA fast track designation for nomacopan for the treatment of hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) in pediatric patients.
HSCT-TMA is an orphan condition that occurs when blot clots form following a stem cell transplant. HSCT-TMA has a fatality rate of more than 80% in children, and has no approved therapies.
“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,” Clive Richardson, Akari’s interim CEO, said in a statement.
Akari plans to commence a pivotal trial in the fourth quarter of 2019 in hopes of expedited approval of nomacopan for HSCT-TMA.