BioBlast Pharma Gets Orphan Drug Designation From EU For Cabaletta - Quick Facts

(RTTNews) - BioBlast Pharma Ltd. (ORPN) said that it has been granted Orphan Drug Designation by the European Commission for Cabaletta for the treatment of Spinocerebellar Ataxia or SCA.

BioBlast is currently conducting a Phase 2 study in patients suffering from SCA3 in Israel.

Cabaletta is a chemical chaperone that has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation, including cell and animal models of Spinocerebellar Ataxia Type 3 (SCA3), Oculopharyngeal Muscular Dystrophy (OPMD), and other PolyA/PolyQ diseases.

BioBlast previously announced that it was starting a Cabaletta Phase 3 study in OPMD in Canada and the U.S.