Shire plc, the global specialty biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has granted marketing approval for VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease in paediatric and adult patients. The FDA designated VPRIV for Priority Review and granted marketing approval in just 6 months. VPRIV offers patients and their physicians a new treatment option at a critical time, as the supply of the previously approved ERT for Gaucher disease is uncertain and remains disrupted.

“We have had the opportunity to use VPRIV in clinical trials and actively participated in the expanded access program. We appreciate the support Shire’s management team has provided during the last few months to ensure continuity of care for nearly 50 of our patients with Gaucher disease. We are confident the team Shire has put into place will ensure a seamless transition into the post-regulatory period,” said Gregory M Pastores associate professor of Neurology and Paediatrics at the NYU School of Medicine in New York. “VPRIV offers patients a therapeutic option that is safe and effective, and our experience with VPRIV has helped build confidence in its use, bolstered by data on low frequency of antibody formation.”

Shire recognizes that the treatments it develops for life-altering diseases and conditions require specialized service and support offerings. With today's FDA approval of VPRIV, the company has implemented enhancements to its existing OnePathSM Access Program with the introduction of a new Co-Pay Assistance Program. The new programme was developed based on feedback from the rare disease community. It is designed to simplify the process and paperwork associated with initiation of therapy, and to reduce the financial burden for patients who are treated with Shire HGT therapies in the United States, including VPRIV. The Company has also announced that it will price VPRIV at a 15% savings over the other commercially available ERT for Gaucher disease.

The new co-pay programme provides assistance for eligible patients in the US who have commercial prescription insurance, and helps these patients pay for out-of-pocket medication costs for Shire HGT products, regardless of income level. Through this program, Shire HGT intends to cover these patients’ insurance co-pay for the first 3 months of their therapy in 2010. In 2011, the Company intends to cap eligible patients’ out of pocket prescription expenses at US$ 500.

The new Co-Pay Assistance Programme will take effect immediately, and will apply to eligible ELAPRASE (idursulfase) patients and VPRIV patients in the US.

“The last six months have been very challenging for the entire Gaucher community, and the approval of VPRIV brings an important new treatment option to patients suffering from Type 1 Gaucher disease,” said Rhonda Buyers, CEO / executive director, National Gaucher Foundation (NGF). “We at the NGF are excited about this approval, and by the steps that Shire has taken to improve access to treatments for patients with life-altering conditions. This co-pay program will greatly assist the Gaucher patient population, and we appreciate the fact that Shire has taken the time to listen to us and to act on the needs of patients.”

The VPRIV application has also been granted accelerated assessment by the European Medicines Agency (EMA) in the European Union (EU). Shire expects to launch VPRIV in the EU by the end of 2010 and in other countries beginning in 2011.

VPRIV (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with Type 1 Gaucher disease.

Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases as well as opportunities in other therapeutic areas to the extent they arise through acquisitions.