Zavesca

Zavesca is the brand name for miglustat, an oral small-molecule drug developed as a substrate reduction therapy for certain lysosomal storage disorders. It inhibits the enzyme glucosylceramide synthase (UGCG), reducing the synthesis of glycosphingolipids and thereby limiting their accumulation within lysosomes. Zavesca is approved in various jurisdictions for the treatment of Niemann-Pick disease type C. In some regions it is indicated for selected patients with Gaucher disease type 1 who cannot receive enzyme replacement therapy or who have contraindications to it.

Administration and dosing are by mouth in capsule form, with the specific regimen determined by regulatory approvals and clinical judgment. Dosing is typically individualized based on tolerability and response, and patients may require dose adjustments to manage adverse effects.

Common adverse effects reported with miglustat include gastrointestinal symptoms such as diarrhea, weight loss, abdominal pain, and flatulence, as well as tremor and peripheral neuropathy in some patients. These effects often emerge early in treatment and may influence adherence or require dose modification. Long-term safety data are limited by small patient populations and the rarity of the underlying diseases.

Safety and monitoring considerations include attention to nutritional status, weight, gastrointestinal tolerance, and neurological symptoms. Contraindications include hypersensitivity to miglustat or to any excipients. Caution is advised in patients with pre-existing neuropathy or significant GI disease, and potential drug interactions should be reviewed by clinicians.

See also Niemann-Pick disease type C; Gaucher disease; substrate reduction therapy.