Shire to supply velaglucerase alfa free for Gaucher disease patients, initially

Velaglucerase alfa, an enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease, may hit the market soon.

Shire plc, the maker of velaglucerase alfa has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency, seeking approval.

Shire plans to provide velaglucerase alfa initially free of charge in order to give suffers of Gaucher.

Shire has already filed marketing applications for velaglucerase alfa in the United States and Canada.

The other drug available to treat Gaucher disease is Cerezyme by Genzyme Corporation which is currently facing problems in production due to an US FDA ban on its manufacturing facility.

Based on a global supply shortage of the currently approved and marketed treatment for patients with Gaucher disease, and positive results from all three velaglucerase alfa Phase III trials, CHMP has accepted the company’s request for an accelerated assessment of the velaglucerase alfa MAA.

The MAA review on velaglucerase alfa is expected to begin in the December cycle.

Under accelerated assessment, the review timeline of the MAA is shortened from 210 days to 150 days.

“Gaucher disease is a debilitating condition and the continuing imiglucerase supply shortage has had a significant impact on patients who have lacked an alternative supply of enzyme therapy,” stated Timothy Cox, M.D., Professor of Medicine at the University of  Cambridge and the founder of the National Centre for the Treatment of Gaucher disease at Addenbrooke’s Hospital, in an official press release.

Shire’s velaglucerase alfa program included over 100 patients at 24 sites in 10 countries around the world have participated in the clinical studies.

Velaglucerase alfa is made using Shire’s proprietary technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and has a human glycosylation pattern.

In Europe and other countries outside the U.S. patients continue to receive velaglucerase alfa through pre-approval access programs that were developed in partnership with national and regional authorities and designed specifically to address the continuing supply shortage. In the U.S., patients continue to be enrolled in an FDA-approved treatment protocol that has been open since September 2009.

What’s Gaucher disease?

Gaucher disease is an enzymatic deficiency caused by autosomal recessive disorder due to mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of glucocerebroside, primarily in macrophages.

In this lysosomal storage disorder (LSD), clinical features are reflective of the distribution of Gaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. The accumulation of glucocerebrosidase in the liver and spleen leads to organomegaly. Bone involvement results in skeletal abnormalities and deformities as well as bone pain crises.

Deposits in the bone marrow and splenic sequestration lead to clinically significant anemia and thrombocytopenia.

Gaucher disease is the most prevalent LSD. Gaucher disease has classically been categorized into 3 clinical types. Type 1 is the most common; it is distinguished from Type 2 and Type 3 by the lack of early neurological symptoms. Type 1 Gaucher disease is characterized by variability in signs, symptoms, severity, and progression.

Headquartered in UK, 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.

Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights.