Sanofi: EU approves drug for Pompe disease

(CercleFinance.com) - Sanofi announced on Tuesday that the European Commission has given marketing authorisation for Nexviadyme, its long-term treatment for late stage and childhood forms of Pompe disease.


The biopharmaceutical group notes that Nexviadyme is the first and only new drug approved for the treatment of Pompe disease in Europe since 2006, when Brussels approved Genzyme's alglucosidase alpha, marketed under the brand name Myozyme.

Pompe disease, a rare, progressive and disabling muscle disorder, is characterised by a deficiency of the lysosomal enzyme acid alpha-glucosidase, which results in the accumulation of glycogen in cells leading to irreversible damage in skeletal muscle and heart muscle.


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