Santhera Prepares for Pivotal Study with Omigapil in Congenital Muscular Dystrophy
Santhera Pharmaceuticals informed about recent achievements and next steps in the clinical development program for omigapil as potential first treatment of Congenital muscular dystrophy (CMD). This severe, genetically determined neuromuscular disease frequently affects infants or young children with life-threatening progressive muscle weakness. The preparation of the further clinical development includes an expert meeting held in Bethesda, Maryland, hosted by the US National Institute of neurological disorders and Stroke (NINDS) and Cure CMD, a dedicated patient advocacy group.
Santhera is currently completing the remaining nonclinical development of omigapil required for the use of the compound in pediatric patients with financial support from the Association Française contre les Myopathies (AFM). Santhera plans to seek protocol assistance and scientific advice from the US Food and Drug Administration and the European Medicines Agency by early 2011. Both agencies have already granted orphan drug designation to the program.
"We would like to thank AFM for their financial support of the nonclinical development for omigapil and Cure CMD and NINDS for organizing this expert meeting in Bethesda. This will be the first pivotal development program ever in this devastating disease. The support of patient advocacy organizations and clinical experts in advancing translational research into the clinic is tremendously important", said Thomas Meier, Chief Scientific Officer of Santhera. "We anticipate the potential initiation of a pivotal study in late 2011 or early 2012."
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