Shire to supply new Gaucher disease drug VPRIV (velaglucerase alfa) through easier access programme

VPRIV (velaglucerase alfa for injection), a human cell line derived enzyme replacement therapy developed by Shire Plc, has been approved for the long-term treatment of Type 1 Gaucher disease in pediatric and adult patients in US.

VPRIV has got a priority review and and US FDA granted marketing approval for the drug in just 6 months.

VPRIV (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term therapy for Type 1 Gaucher disease

VPRIV was studied in three clinical trials in a total of 99 patients with type 1 Gaucher disease.

Eighty-two patients age 4 years and older received VPRIV and 17 patients age 3 years and older received imiglucerase.

The first two studies were conducted in patients who were not currently receiving Gaucher disease-specific therapy.

Study III was conducted in patients who were receiving imiglucerase treatment immediately before starting VPRIV.

VPRIV was administered intravenously over 60 minutes at doses ranging from 15 Units/kg to 60 Units/kg every other week. Each study met its primary endpoint.

What is Gaucher disease?

Gaucher disease is an autosomal disorder. It is caused by mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase.

Deficiency of beta-glucocerebrosidase causes an accumulation of glucocerebroside, primarily in macrophages.

Gaucher disease is the most prevalent of the lysosomal storage disorders diseases. Clinical features of this lysosomal storage disorder are reflective of the distribution of Gaucher cells in the liver, spleen, bone marrow, and other organs.

The accumulation of glucocerebrosidase in the liver and spleen leads to organomegaly.

Presence of Gaucher cells in the bone marrow and spleen lead to clinically significant anemia and thrombocytopenia.

Gaucher disease has been categorized into 3 clinical types. Type 1 is the most common and is distinguished from Type 2 and Type 3 by the lack of early neurological symptoms.

Shire plc has implemented OnePath Access Program with the introduction of a new Co-Pay Assistance Program.

The new program 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.

Shire plans to price VPRIV at a 15% savings over the other commercially available enzyme replacement therapy for Gaucher disease.

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, Shire 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 (idursulfase) patients and VPRIV patients in the U.S.

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.

Shire Plc is a specialty biopharmaceutical company. Shire focuses on deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases.

Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets.