Pfizer's Elelyso For Gaucher Disease Approved By FDA

Pfizer and its partner Protalix Biotherapeutics announced that the U.S. Food and Drug Administration (FDA) has approved its Elelyso (taliglucerase alfa) for injection for the treatment of type 1 Gaucher Disease.

Elelyso was previously approved for the treatment of pediatric patients. The new approval allows Elelyso to be used as treatment for long-term enzyme replacement therapy (ERT) for both adult and pediatric patients diagnosed with Type 1 Gaucher disease.

Rory O’Connor, SVP of Global Medical Affairs at Global Innovative Pharma Business, Pfizer, said, “The approval of ELELYSO to treat pediatric patients with Type 1 Gaucher disease provides physicians another treatment option for this rare and potentially debilitating disease. This pediatric indication, along with the recent announcement that ELELYSO received kosher certification by the Orthodox Union (OU), reinforces the ongoing commitment of Pfizer to addressing the needs of the Gaucher community.”

Gaucher disease is an inherited lysosomal storage disorder that causes severe and debilitating symptoms, including anemia, liver and spleen enlargement, and various bone disease. Type 1 Gaucher disease is the most common type of the disease, estimated to affect 10,000 people worldwide.

“While Type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood. It is important that children with this disease have access to a range of FDA-approved treatment options that are effective,” said Paige Kaplan, Section of Biochemical Genetics (Metabolic Diseases), Children's Hospital of Philadelphia.

The approval was supported by positive results from the 12-month randomized, double blind, multi-center study in patients with Gaucher disease who have not yet undergone treatment. Results show that Elelyso achieved a decrease in spleen and liver volume of patients, as well as increased platelet count.

Last month, another treatment got the green light for treatment of the rare disease. Sanofi received approval from the FDA for its Cerdelga pill for Gaucher disease. The new approval supplements the company’s 20-year old drug for Gaucher disease, the intravenous infusion Cerezyme.