OREANDA-NEWS. Santhera Pharmaceuticals (SIX: SANN) announces that it has received Fast Track Designation from the US Food and Drug Administration (FDA) for omigapil for the treatment of congenital muscular dystrophies (CMD). FDA's Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need for the purpose of getting them to the patient earlier. Previously, omigapil has already been granted Orphan Drug Designation for CMD in both the EU and the US. Santhera, in collaboration with the US National Institutes of Health (NIH), is currently conducting a clinical phase I study (CALLISTO) with omigapil in CMD patients.

"We are delighted that the FDA has granted Fast Track Designation for omigapil thereby underlining the unmet medical need for an effective therapy for CMD patients and the severity of this class of diseases," commented Thomas Meier, PhD, CEO of Santhera. "After Raxone®, omigapil is our second neuromuscular pipeline product and underpins our dedication to developing effective medicines to satisfy the needs of patients suffering from mitochondrial and other rare diseases."

CMD is a group of inherited, severe neuromuscular diseases characterized by different forms of progressive and ultimately devastating loss of muscle tissue, frequently affecting young children. Currently, no treatment is available to slow down or stop progression of the disease. Santhera evaluates omigapil in the phase I study (CALLISTO) which investigates the safety, tolerability and pharmacokinetic profile of a new liquid formulation of omigapil in pediatric and adolescent patients with CMD. The study, which is being conducted at the US NIH's National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland, with Prof. Carsten Bönnemann as Principal Investigator, is fully enrolled and expected to be completed early in 2017. As previously reported, the CALLISTO study is supported financially by a public-private partnership including two patient organizations, the US-based Cure CMD and the Swiss Foundation for Research on Muscle Diseases, and EndoStem, an EU 7th Framework program.