Akari's nomacopan designated orphan drug by FDA for HSCT-associated thrombotic microangiopathy

Shares in Akari Therapeutics jumped as much as 13% on Friday after the company said its investigational therapy nomacopan, formerly known as Coversin, received an orphan drug designation from the FDA for the treatment of haematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA). 

The US regulator recently fast-tracked nomacopan for the same indication in paediatric patients. Earlier this year, Akari reached an agreement with the agency regarding the framework of the company's proposed pivotal clinical trial programme for paediatric HSCT-TMA. Akari said a pivotal trial for the therapy in HSCT-TMA is slated to start in the fourth quarter. 

CEO Clive Richardson stated that receiving the orphan drug and fast-track designations for nomacopan "underscores the significant unmet medical need in this disease...for which there are currently no approved treatments." He added "we look forward to taking advantage of the opportunities that [these designations] provide across all stages of drug development in order to bring this potential new treatment option to patients as rapidly as possible."

Akari explained that nomacopan is a C5 complement inhibitor that also inhibits leukotriene B4. Aside from thrombotic microangiopathies, the treatment is also under development for use in patients with bullous pemphigoid, atopic keratoconjunctivitis and paroxysmal nocturnal haemoglobinuria.

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