Bio Blast: Cabaletta Gets Orphan Drug Designation For Treatment Of SCA3 Disease

(RTTNews) - Bio Blast Pharma Ltd. (ORPN) announced it has been granted Orphan Drug Designation by the U.S. FDA for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (known as SCA3 and Machado Joseph disease). This is the second indication for which Bio Blast's Cabaletta has received such designation.

Cabaletta is a chemical chaperone that protects against pathological processes in cells. The company noted that Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Occulopharyngeal Muscular Dystrophy and Spino bulbar cerebellar ataxia. Bio Blast plans to make clinical progress in each of these indications in 2015.