Shire's Gaucher disease drug enters fast-track EU approval process

Britain's biopharmaceuticals group Shire is racing to end a shortage of drugs to treat Type 1 Gaucher disease, a condition caused by an enzyme deficiency that can cause neurologic problems, skeletal disorders and liver malfunction. Shire has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for its Gaucher drug velaglucerase alfa, an enzyme replacement therapy.

This follows positive results in all three of Shire's phase III trials, which were the largest and most comprehensive to date for Gaucher disease, says Shire. Over 100 people at 24 sites in 10 countries participated in them. The MAA was also spurred by a global supply shortfall of the only drug hitherto approved for Guacher disease, Genzyme Corp's Cerezyme. Genzyme has been hit by trouble at a manufacturing site in Boston, where contamination of a bioreactor with a virus hampered Cerezyme production. As a result, the EMA's Committee for Medicinal Products for Human Use has granted Shire's request for an accelerated assessment of velaglucerase alfa. That shortens the review from 210 to 150 days. America's Food and Drug Administration also recently agreed to fast-track Shire's application for approval.

At present, velaglucerase alfa is being supplied to patients in Europe and outside the US through pre-approval access programmes developed by Shire with national and local authorities in an effort to tackle the supply squeeze. In America, there is an FDA-approved treatment protocol that has been available to patients for the past two months.