Akari Therapeutics PLC (NASDAQ: AKTX) shares jumped on Wednesday after the firm announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its stem cell transplant treatment.
Specifically, the designation is for nomacopan for the treatment of hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) in pediatric patients.
Overall, 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.
HSCT-TMA is an orphan condition with an estimated fatality rate of more than 80% in children with the severe disease. There 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.
Clive Richardson, interim CEO of Akari Therapeutics, commented:
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.
Shares of Akari traded up about 30% to $2.36 early Wednesday. The 52-week trading range is $1.56 to $9.20.