FDA Grants Orphan Drug Status To Bio Blast's Cabaletta For Spinocerebellar Ataxia Type 3
By Cyndi Root
Bio Blast Pharma announced in a press release that the Food and Drug Administration (FDA) has granted Orphan Drug status to Cabaletta in the treatment of Spinocerebellar Ataxia type 3, the second Orphan designation for Cabaletta. The company is conducting Phase 2 and Phase 3 trials for several indications.
Dalia Megiddo, MD, CEO of Bio Blast, said, "This orphan drug designation lays the foundation for what we believe are very favorable conditions to continue to pursue our clinical plans. 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."
Cabaletta
Cabaletta works against pathological processes in cells by acting as a chemical chaperone. The agent is effective in several diseases because it prevents proteins from aggregating in a pathological or abnormal way. In preclinical studies and animal models, the agent shows promise in treating SCA3, Occulopharyngeal Muscular Dystrophy (OPMD), and Spino bulbar cerebellar ataxia (SBMA).
Spinocerebellar Ataxia Type 3
Spinocerebellar Ataxia type 3 is a movement disorder. Patients have early symptoms of coordination and balance deficits, difficulty speaking, muscle stiffness, and double vision. Sleep disorders are prevalent, leaving patients tired during the day. When the disease progresses, patients lose sensations, feel weak in the limbs, and have cognitive difficulties. Eventually, most patients require wheelchairs and die 10 to 20 years after symptoms appear.
Bio Blast Pharma
Bio Blast Pharma is a publicly traded, clinical-stage biotechnology company that was founded in 2012. Headquartered in Israel, the company focuses on understanding biological mechanisms and pathways in order to find treatments for several diseases that share similarities. In September 2014, the company announced that Health Canada granted approval to begin enrolling patients for the HOPEMD Phase 2/3 study. The trial is evaluating Cabaletta in OPMD at the Montreal Neurological Institute at McGill University.
In August 2014, the United States Patent and Trademark Office (USPTO) notified Bio Blast of a “notice of allowance,” signifying that the agency believes the company is entitled to a patent. The composition-of-matter patent is for Bio Blast’s novel fusion protein for Friedrich’s Ataxia built on the company’s mitochondrial protein replacement (mPRT) platform.