Shire receives marketing approval from FDA for VPRIV

Feb 27 2010

Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the U.S. 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 pediatric 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 MD, Associate Professor of Neurology and Pediatrics 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 program 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.

New Co-Pay Assistance Program for Eligible U.S. Patients

The new co-pay program provides assistance for eligible patients in the U.S. 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 $500.

The new Co-Pay Assistance Program will take effect immediately, and will apply to eligible ELAPRASE(R) (idursulfase) patients and VPRIV(TM) patients in the U.S.

"The last 6 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."

SOURCE Shire Pharmaceutical