Santhera's Omigapil Receives Orphan Drug Designation from FDA and EMEA

18-Aug-2008

Santhera Pharmaceuticals announced that the US food and Drug Administration (FDA) has granted Orphan Drug Designation to SNT-317/omigapil for treatment of Congenital muscular dystrophy. This severe, genetically determined neuromuscular condition frequently affects infants or young children with life-threatening progressive muscle weakness. The decision from the FDA follows shortly after the European Medicines Agency (EMEA) granted orphan drug designation for the two most common subtypes of the disease.

In 2007, Santhera licensed omigapil from Novartis for development in Congenital Muscular Dystrophy and other neuromuscular diseases. Tests in a disease-relevant model have shown that SNT-317/omigapil prevents apoptosis and ameliorates the pathology of laminin-alpha 2 deficient muscular dystrophy. Preclinical data will be presented at the upcoming 13th International World Muscle Society Congress in Newcastle, United Kingdom.

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