(Reuters) -The U.S. Food and Drug Administration has asked for a resubmission of Stealth BioTherapeutics’ marketing application for its experimental therapy targeting a rare genetic condition, Barth syndrome, the company said on Thursday.
The drug developer added that it has implemented a 30% workforce reduction to conserve resources for a potential resubmission.
The federal health regulator’s decision marks another setback in Stealth’s prolonged effort to bring the therapy, elamipretide, to market after several delays.
Stealth received the FDA’s so-called ‘complete response letter’ after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline.
The Needham, Massachusetts-based company is seeking approval for elamipretide, a potential treatment for Barth syndrome – a condition that typically affects boys and causes heart, muscle weakness and delays growth.
Stealth had submitted data from a mid-stage trial, which showed improvement in knee muscle strength, which was correlated with improvements on the six-minute walk test.
The FDA has agreed to consider knee extensor muscle strength, for which the company previously submitted data, as a goal to support accelerated approval, the company said.
However, the regulator is hesitant to extend the accelerated pathway to critically ill newborns, who make up nearly two-thirds of the therapy’s expanded access program participants.
Barth syndrome affects fewer than 150 individuals in the United States and fewer than 300 worldwide, and currently, no approved therapies exist.
(Reporting by Sriparna Roy in Bengaluru; Editing by Tasim Zahid)
Comments