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FDA approves taliglucerase alfa for treatment of Gaucher's disease
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The FDA recently approved taliglucerase alfa for long-term enzyme replacement therapy to treat a form of the rare genetic disorder Gaucher’s disease, according to a press release.
Taliglucerase alfa (Elelyso, Pfizer) is an injection that replaces the missing enzyme in patients with a confirmed diagnosis of type 1 (non-neuropathic) Gaucher’s disease and should be administered by a health care professional every other week.
Gaucher’s disease occurs in those who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes lipids to collect in the spleen, liver, kidneys and other organs. Major signs include liver or spleen damage, anemia, low blood platelet counts and bone problems.
“[This approval] provides for a new enzyme replacement therapy for the select number of patients with type 1 Gaucher disease,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research. “It also demonstrates FDA’s commitment to developing treatments for rare diseases.”
Because the disease affects a small number of people, researchers evaluated the drug’s efficacy in 56 patients enrolled in two clinical trials. Many of these patients continued treatment on a longer-term extension study.
In one multicenter, double blind, parallel-dose trial, researchers evaluated taliglucerase alfa as an initial therapy in 31 adult patients who had not previously received enzyme replacement therapy. They randomly assigned patients to receive a 30-U/kg dose or 60-U/kg dose.
Results indicated that taliglucerase alfa was effective in reducing spleen volume — the study’s primary endpoint. Patients assigned 30 U/kg and those assigned 60 U/kg experienced 29% and 40% reductions in spleen volume, respectively, after 9 months of treatment. The researchers also observed improvements in liver volume, blood platelet counts and hemoglobin levels.
In another multicenter, open-label, single-arm trial, researchers assessed the drug’s efficacy in 25 patients with type 1 Gaucher’s disease who were switched from imiglucerase (Cerezyme, Genzyme), another enzyme replacement therapy product, to taliglucerase alfa. All patients had been receiving treatment with imiglucerase for at least 2 years before switching to taliglucerase alfa infusions every other week at the same dose of imiglucerase. Results showed that taliglucerase alfa was effective in maintaining spleen and liver volumes, blood platelet counts and hemoglobin levels during a 9-month evaluation period.
The most common adverse effects were infusion reactions and allergic reactions. Symptoms of infusion reactions include headache, chest pain or discomfort, weakness, fatigue, hives, skin, redness, increased blood pressure, back pain, joint pain and flushing. As with other IV protein products, anaphylaxis has been observed in some patients.
Other common adverse effects observed in more than 10% of patients include upper respiratory tract infection, nasopharyngitis, arthralgia, influenza, headache, extremity pin, back pain and urinary tract infections.
Elelyso is manufactured and distributed by Pfizer under license from Protalix Biotherapeutics Inc.