Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion on the marketing authorisation for VPRIV™ (velaglucerase alfa), the company’s enzyme replacement therapy (ERT) for the treatment of type 1 Gaucher disease. The CHMP positive opinion will now be forwarded to the European Commission for ratification, and marks a significant milestone in bringing a new Gaucher treatment to market in all EU Member States.
"Gaucher disease is a rare and often debilitating condition," said Professor Timothy Cox, Department of Medicine, University of Cambridge based at Addenbrookes Hospital UK. "I am pleased that, given the difficulties in supplying enzyme treatment for our patients over the last year, we will soon have the opportunity to prescribe velaglucerase alfa without regulatory constraints; the treatment represents an important option in this therapeutic arena."
In many European countries patients have been receiving VPRIV on an early access named patient basis, developed in partnership with national and regional authorities. These programs, designed specifically to address the continuing supply shortage of the only other commercially marketed ERT, have experienced strong uptake from physicians and patients and have garnered the support of treatment working groups and advocacy organisations. To date, hundreds of patients in the EU have been treated with VPRIV through Shire’s clinical trials or early access mechanisms.
“A positive opinion for VPRIV in the EU, earlier than expected, is very good news, particularly as we have been working with physicians in 22 countries for almost a year to treat patients with type 1 Gaucher disease through early access programs,” said Sylvie Grégoire, President, Shire Human Genetic Therapies. “We look forward to our ongoing collaboration with these physicians and the Gaucher community at large as we enter the final stages of the approval process in Europe.”
In addition to the CHMP positive opinion, VPRIV has received orphan drug designation from the Committee for Orphan Medical Products.
With the accelerated adoption of VPRIV worldwide, and the earlier than anticipated US approval and EU positive opinion, Shire expects continued high demand for the therapy. As a result of this, the company is now implementing a program with physicians and patients to monitor and manage requests from new patients carefully in order to ensure long-term, uninterrupted treatment with VPRIV.
Shire’s VPRIV clinical development program represents the largest and most comprehensive clinical data set supporting registration for an ERT for Type 1 Gaucher disease to date.