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.
"With the grant announced today, we have now received Orphan Drug Designation by both the European Commission and the U.S. FDA for Cabaletta in two diseases: Spinocerebellar Ataxia Type 3 (SCA3) and Oculopharyngeal Muscular Dystrophy (OPMD). This is yet another important step in the development of this drug candidate," stated Colin Foster, President and Chief Executive Officer of BioBlast.
SCA3, also known as Machado Joseph disease, is the most common disease among the cerebellar ataxias, which are a group of genetic diseases that are characterized by memory deficits, spasticity, difficulty with speech and swallowing, weakness in arms and other muscular disorders.
Symptoms can begin in early adolescence and get worse over time. Eventually SCA3 leads to paralysis, and severe cases can lead to an early death in the fourth decade of life. SCA3 is incurable, and there is currently no approved treatment for the disease.