BioBlast Pharma Ltd., a clinical-stage biotechnology firm focused on orphan disease treatment solutions, recently announced that its intravenous (IV) solution Cabaletta received Fast Track designation from the United States Food and Drug Administration (FDA) to address Oculopharyngeal Muscular Dystrophy (OPMD) patients.
Cabaletta is being advanced to address treatment to OPMD, which is an uncommon and debilitating form of muscular dystrophy with no approved therapies approaches or cure. The company is currently launching a Phase ⅔ clinical study for the disease and recently announced the granting of an Investigational New Drug (IND) that enables the opening of a United States-based clinical center to be added to the two currently ongoing clinical sites in Canada and Israel.
OPMD is an inherited myopathy that is characterized by dysphagia or troubles with swallowing and by the loss of strength in the muscles. When dysphagia becomes more severe, patients lose significant weight and become malnourished, dehydrated and might develop pneumonia. The cause of the disease is a genetic mutation that results in an unstable protein (PABPN1) that clumps inside patients’ muscle cells.
Cabaletta, one of the many BioBlast Pharma’s candidates, has been proved to prevent this aggregation of proteins and has been documented as evidencing high efficacy in pre-clinical animal models of OPMD.
The Fast Track program is established under the 1997’s FDA Modernization Act, designed to accelerate the advancement and the reviewing of drugs that are intended to address serious conditions and stuff relevant unmet medical need. If a drug is included in a development program with the Fast Track stamp, greater access to the FDA is granted in order to accelerate the drug’s development and review and its chances of approval.
“We believe that the Fast Track designation represents an important recognition by the FDA of Cabaletta’s potential to address a significant unmet need in the treatment of OPMD patients. We will continue to work closely with the FDA with the goal of bringing Cabaletta to OPMD patients as quickly as possible,” said Colin Foster, the BioBlast’s Chief Executive Officer and President in a press release.
Bioblast Pharma’s website: www.bioblast-pharma.com