Santhera licenses Omigapil for treatment of rare neuromuscular disorders

Santhera Pharmaceuticals, a Swiss based company with special focus on the development of treatment for neuromuscular disorders, announced on Monday a licensing agreement with Novartis to test their compound omigapil for the treatment of Congenital muscular dystrophy

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What is Congenital muscular dystrophy (CMD)?

Congenital muscular dystrophy is a group of rare inherited disorders in which symptoms appear at birth or within the first six month of life. It is characterised by progressive loss of muscle tissue and individuals appear 'floppy' with a low muscle tone and contractures. The severity of the different forms is clinically and genetically very diverse and clinical features can range from relatively mild with survival into adult life to very severe and devastating with reduced life expectancy. There is currently no effective treatment or cure available, but orthotics, physiotherapy and ventilation can help to alleviate the symptoms.

How might the compound omigapil treat CMD?

The compound omigapil interacts with an enzyme that is known to be involved in cell death. In preclinical trials carried out in animal models for CMD, Santhera could show that omigapil prevents loss of muscle tissue and imcreases body weight and survival.

The agreement with Novartis offers Santhera the possibility to develop omigapil as a potential treatment for CMD, but also covers the option to expand the development into treatments for other neuromuscular disorders.

What stage is the treatment currently at?

Santhera anticipates starting clinical trials before the end of 2008, but also states that, given its intended use in children, additional preclinical work requires to be carries out before commencing a Phase II clinical trial in CMD patients.


Further information and links

About Congenital muscular dystrophy
For more information about this condition, go to our information pages

About Santhera
For more information about Santhera's work on omigapil and CMD, go to Santhera's website www.santhera.com.