Shire announced it has received the approval of the U.S. Food and Drug Administration (FDA) for the production of its VPRIV drug substance (velaglucerase alfa for injection). The drug substance will be manufactured at Shire’s facility at 400 Shire Way, Lexington, Massachusetts. Bill Ciambrone, Executive Vice President, Technical Operations at Shire, said, “Shire has always been committed to providing uninterrupted treatment for all VPRIV patients at the dose and frequency prescribed by their physicians. We continue to deliver on this commitment.”
The company invested more than $200 million in technology and manufacturing infrastructure to provide a stable drug supply chain to patients using VPRIV. Shire’s new 400 Shire Way facility is the first commercially licensed facility worldwide to utilize single-use bioreactor and disposable technology throughout cell culture processing built and designed to reduce manufacturing risk.
VPRIV is intended for the long term treatment of patients with type 1 Gaucher disease. The drug substance is made in a human cell using the company’s gene activation technology. The resulting enzyme contains the exact human amino acid sequence found in the naturally occurring human enzyme. VPRIV is indicated in patients previously treated for type 1 Gaucher disease or those who are treatment-naïve. The drug substance has been granted regulatory approval in more than 40 countries worldwide including the U.S., the European Union member states and Israel.
Gaucher disease is the most common lysosomal storage disorder in which the body is unable to break down glucocerebroside. The disease can affect various parts of the body including the spleen, liver, and bone marrow.
Rhonda Buyers, CEO and Executive Director, National Gaucher Foundation said, “FDA approval of Shire's manufacturing facility in Lexington provides greater assurance that Gaucher patients will receive consistent and uninterrupted access to enzyme replacement therapy for the treatment of type 1 Gaucher disease.”
With the approval of the Lexington facility, Shire now has two FDA and EMA approved facilities (the other is located in Alewife) to manufacture VPRIV drug substance.