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FDA Approves Oral Capsules for Gaucher's Disease Treatment

Kate Traynor

The Food and Drug Administration (FDA) on Thursday approved the marketing of miglustat oral capsules for the treatment mild to moderate type 1 Gaucher's disease in adults for whom enzyme replacement therapy is not a therapeutic option.

According to the product's manufacturer, Switzerland-based Actelion Ltd., miglustat is the first FDA-approved oral therapy for patients with type 1 Gaucher disease, which affects about 10,000 people worldwide. Miglustat will be available in the United States later this year under the brand name Zavesca.

Type 1 Gaucher's disease is a genetic disorder caused by a deficiency of glucocerebrosidase and characterized by the excess accumulation of lipids in storage cells in the liver, spleen, lymph nodes, alveolar capillaries, and bone marrow. Symptoms of the disorder include spleen and liver enlargement, anemia, and bone pain. Intravenous enzyme replacement therapy is the first-line treatment for the disease.

According to Actelion, miglustat inhibits glucosylceramide synthase, an enzyme that is required for the synthesis of most glycolipids, including those metabolized by glucocerebrosidase. The product is classified as a substrate reduction therapy, reportedly the first drug in this class for use by patients with Gaucher's disease.

Although the FDA-approved labeling for miglustat is not yet available, information about the product appears in labeling approved for marketing Zavesca in the European Union (EU). According to the EU-approved labeling, adverse events reported by at least 10 percent of miglustat users were: weight loss; visual disturbance; diarrhea and other gastrointestinal symptoms; and nervous-system symptoms such as tremor, dizziness, headache, and leg cramps.

The EU-approved labeling states that all patients should undergo periodic screening for symptoms of peripheral neuropathy before and periodically during treatment with miglustat. The labeling also recommends baseline and repeat cognitive function testing of patients who use the drug, which has been associated with "isolated cases of cognitive dysfunction."

Miglustat is marketed in the EU as 100-mg hard capsules. The recommended dosage is 100 mg three times a day, taken with or without food. A lower dosage, 100 mg once or twice daily, may be needed for patients who suffer from diarrhea while taking the product.