Gaucher Disease Treatment Therapy Approved

Velaglucerase alfa for injection (VPRIV™) has been approved  by the U.S. Food and Drug Administration (FDA) for the treatment Gaucher disease—a rare genetic disorder that affects both children and adults.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this enzyme, harmful amounts of a certain fatty substance (lipid) can build up in the liver, spleen, bones, bone marrow, and nervous system, and can prevent cells and organs from working properly. About one in 50,000-100,000 people in the general population have Gaucher disease.

VPRIV provides long-term enzyme replacement therapy (ERT) for Type 1 Gaucher disease, the most common form of the genetic disorder. It is an alternative to Cerezyme (imiglucerase), another enzyme replacement therapy, which is currently in short supply.

“The approval of VPRIV will provide a safe and effective alternative treatment for patients with Gaucher disease,” said Julie Beitz, M.D., director of the FDA’s Office of Drug Evaluation III, in a press release. “Patients who previously received Cerezyme as an enzyme replacement therapy for their Type 1 Gaucher disease can be safely switched to VPRIV.”

Indications and Usage

VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term ERT for pediatric and adult patients with Type 1 Gaucher disease.

Dosing and Administration

Shire plc, the maker of VPRIV, is recommending the following dosing and administration guidelines:

  • 60 units/kg administered every other week as a 60-minute intravenous infusion.
  • Patients currently being treated with imiglucerase for Type 1 Gaucher disease may be switched to VPRIV. Patients previously treated on a stable dose of imiglucerase are recommended to begin treatment with VPRIV at that same dose when they switch from imiglucerase to VPRIV.
  • Physicians can make dosage adjustments on an individual patient basis based on achievement and maintenance of therapeutic goals. Clinical trials have evaluated doses ranging from 15 units/kg to 60 units/kg every other week.

Co-Pay Assistance

Shire’s new Co-Pay Assistance Program assists eligible U.S. patients who have commercial prescription insurance pay for out-of-pocket medication costs for Shire HGT products, regardless of income level. Through this program, Shire said it will cover patients’ insurance co-pay for the first three months of their therapy in 2010. In 2011, the company intends to cap eligible patients’ out-of-pocket prescription expenses at $500.

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