FDA Approves Taliglucerase Alfa for Gaucher Disease

Emma Hitt, PhD

May 02, 2012

May 2, 2012 — The US Food and Drug Administration (FDA) has approved the orphan drug taliglucerase alfa (Elelyso, Protalix BioTherapeutics Inc) as a long-term enzyme replacement treatment for type 1 (non-neuropathic) Gaucher disease.

The FDA announced the approval yesterday in a news release. Two other enzyme replacement therapies, imiglucerase and velaglucerase, are approved for Gaucher disease in the United States.

The efficacy of taliglucerase alfa was evaluated in 56 patients with type 1 Gaucher disease participating in 2 multicenter clinical trials.

One trial evaluated efficacy in 31 adult patients who had not previously received enzyme replacement therapy. Patients were randomly assigned to receive taliglucerase alfa at a dose of 30 U/kg or 60 U/kg, administered by intravenous infusion every other week.

Taliglucerase alfa reduced spleen volume, the study’s primary endpoint, at both doses. At 9 months, spleen volume was reduced by an average of 29% and 40% with the lower and higher doses, respectively.

Treatment also resulted in reduced liver volume and increased blood platelet counts and hemoglobin levels.

The other study included 25 patients who had already been receiving imiglucerase as enzyme replacement therapy for at least 2 years. In this trial, taliglucerase alfa was administered at the same dose of imiglucerase, after imiglucerase was stopped.

As with the first study, taliglucerase alfa maintained reduced spleen and liver volumes, blood platelet counts, and hemoglobin levels for 9 months.

Adverse events associated with taliglucerase alfa included infusion reactions and allergic reactions, with some instances of anaphylaxis. In addition, more than 10% of patients experienced upper respiratory tract infection, nasopharyngitis, arthralgia, influenza, headache, extremity pain, back pain, and urinary tract infections.

According to a press release from Pfizer, which will distribute the new therapy under license from Protalix BioTherapeutics Inc, supply disruptions of approved enzyme replacement therapies have been affecting patients living with Gaucher disease since 2009 in multiple countries, including the United States.

The company says it will attempt to maintain a continuously restocked 24-month supply at various stages of production for US patients prescribed taliglucerase alfa.

"Taliglucerase alfa for injection will be available to US patients at a cost that will be priced at 25% below the cost of imiglucerase," the company states.

Gaucher disease results from a deficiency in glucocerebrosidase, causing the accumulation of lipids in various organs, including the spleen, liver, and kidneys. Type 1 Gaucher disease affects about 6000 people in the United States.


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