Omigapil is a deprenyl analogue with anti-apoptotic properties in development for the treatment of congenital muscular dystrophy (CMD).1–3
CMD refers to a variety of inherited neuromuscular conditions characterized by different forms of progressive loss of muscle tissue.4–7 Severe forms can affect newborns or young children with life-threatening progressive muscle weakness (“floppy infant syndrome”).1,2,4 Complications associated with the disorder such as loss of body weight, skeletal deformations and respiratory distress result in immobility at a young age and early mortality.4–8
No pharmacological therapy is currently available or in advanced clinical development for CMD.6,9 Treatment options are confined to respiratory support and orthopedic surgery as well as supplementary nutrition to avoid malnutrition.9
In preclinical research, Santhera demonstrated that omigapil prevents apoptosis and loss of muscle tissue, ameliorates muscle histology and increases body weight and survival of a disease-relevant animal model for CMD.1
Santhera has in-licensed omigapil for development and commercialization in CMD, and initiated a clinical development program with public–private partners. Omigapil has been granted orphan drug designation for CMD in Europe and the US.
The Phase I study (CALLISTO) is evaluating the pharmacokinetic profile, safety and tolerability of oral omigapil in pediatric and adolescent CMD patients and will establish the feasibility of conducting disease-relevant clinical assessments for the design of future efficacy trials.2
Erb M, et al. J Pharmacol Exp Ther 2009; 331:787–795.
Erb M, et al. PLoS One 2012; 7:e36153.
Clinical Trials Identifier NCT01805024 (CALLISTO trial). Available here. Accessed December 2015.
Bertini E, et al. Semin Pediatr Neurol 2011; 18:277–288
Lisi MT & Cohn RD. Biochim Biophys Acta 2007; 1772:159–172
Muscular Dystrophy Campaign UK: Congenital Muscular Dystrophies Fact Sheet. Available here.
Bönnemann CG, et al. Neuromuscul Disord 2014; 24:289–311.
Wang CH, et al. J Child Neurol 2010; 25:1559–1581.