The European Commission has granted marketing authorisation to Shire’s Vpriv for the long-term treatment of type 1 Gaucher disease clearing the drug’s use in 30 countries across the region.

Vpriv (velaglucerase alfa for injection) is a human cell line derived enzyme replacement therapy (ERT) designed for use in both adult and paediatric patients, which has, in extensive clinical trials, proven its potential as a long-term therapy for sufferers of the disease.

Gaucher disease is a rare and often debilitating lysosomal storage disorder caused by a hereditary deficiency of the enzyme glucocerebrosidase, which can lead to the build up of fatty substances in organs including the spleen, liver, kidney and brain, resulting in a whole host of complications such as liver malfunction, skeletal disorders anaemia.

Vpriv’s European approval is important because it allows an alternative, licensed therapeutic enzyme to treat the disease, said Professor Tim Cox from the Department of Medicine, University of Cambridge, and founder of the largest Gaucher clinic in the UK. “For type 1 patients the availability of Vpriv provides further opportunities to individualise treatment options for this complex disorder,” he explained.

Shire has been providing its ERT to patients free of charge on both sides of the Atlantic through early access programmes - which allow patients access to a drug before it becomes commercially available under certain conditions - following a global supply shortage of Cerezyme after viral contamination forced Genzyme to temporarily shut down a manufacturing plant last June.

A subsequent ramp up in manufacturing, clinical and regulatory timelines for Vpriv has resulted in its regulatory approval “months ahead of schedule”, Shire said.