Press release of 19 november 2014
BioBlast Pharma announced today that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (SCA3, Machado Joseph disease).
Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation. Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Oculopharyngeal Muscular Dystrophy (OPMD) and Spino bulbar muscular atrophy. BioBlast plans to make clinical progress in each of these indications in 2015.
"The orphan drug designation is yet another step on BioBlast’s route to create and capture value from our research and clinical work,” stated Dalia Megiddo, MD MBA, Chief Executive Officer of BioBlast. “We currently are conducting a phase 2 clinical trial to test the efficacy of Cabaletta in SCA3 and plan to start our SCA3 pivotal study in 2015.”
About Orphan Drug Designation
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waives the Prescription Drug User Fee Act (PDUFA) filing fees.
About BioBlast Pharma
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology. For more information please visit the Company’s website, www.bioblast-pharma.com, the content of which is not incorporated herein by reference.
