Actelion announces positive opinion for miglustat

Actelion said today that the Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion to the extension of indication for miglustat (Zavesca).

This refers to the drug used in those with Niemann-Pick type C (NPC), a neurodegenerative genetic disease – a lipid storage condition that can affect infants, youngsters and adults.

Zavesca (100 mg miglustat) is already indicated for oral treatment in patients with mild to moderate type 1 Gaucher disease where enzyme replacement therapy cannot be administered.

Today’s development means the European Commission is expected to follow the CHMP’s opinion and make a final decision within two months.

In July of this year, Actelion announced it was to collaborate with GlaxoSmithKline (GSK) for almorexant, an orexin receptor antagonist for the treatment of primary insomnia and one in phase III development.

The agreement involved GSK receiving global exclusive rights for the co-development and co-commercialisation of the drug, and contributing 40 per cent of the costs.

“By targeting orexin, which is known to help regulate the sleep-wake cycle, these novel molecules could help to reduce or even eliminate some of the side-effects associated with current sleep treatments,” explained Dr Moncef Slaoui, GSK’s chairman of research and development.