MITO - Stealth Bio climbs on FDA's Orphan Drug status for lead asset in muscular disorder

Stealth BioTherapeutics (NASDAQ:MITO) shares have surged ~52% in the pre-market Thursday after the clinical-stage biotech announced that the FDA granted its Orphan Drug Designation for its lead asset elamipretide as a treatment for patients with Duchenne muscular dystrophy. In addition, Stealth (MITO) said that the FDA’s Division of Neurology accepted the company’s request for a pre-IND meeting on the development of elamipretide in combination with an approved drug class called exon-skipping phosphorodiamidate morpholino oligomers (PMO). With its orphan drug designation, the FDA aims to offer financial incentives to drug developers targeting rare diseases and conditions. In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity upon regulatory approval of the treatment.

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Stealth Bio climbs on FDA’s Orphan Drug status for lead asset in muscular disorder