European Commission Grants Orphan Drug Status For Cabaletta To Treat OPMD

(RTTNews) - BioBlast Pharma Ltd (ORPN), a clinical-stage biotechnology company, on Thursday said the European Commission has granted Orphan Drug Designation for Cabaletta to treat Oculopharyngeal Muscular Dystrophy ("OPMD").

BioBlast said it is starting a double-blind, placebo-controlled Phase 3 pivotal study across clinical centers in the US and Canada in OPMD.

OPMD is a rare inherited myopathy characterized by dysphagia (difficulty in swallowing), the loss of muscle strength, and weakness in multiple parts of the body. There is no medical treatment or, to our knowledge, potential cure for OPMD.