ZAVESCA

What it is

ZAVESCA is miglustat 100 mg capsules. It is presented as hard white capsules with "OGT918" printed in black on the cap and "100" printed on the body. Miglustat is an orally active, non-peptide, N-alkylated imino sugar, which is a synthetic analogue of D-glucose.

Approved indications

— Oral treatment of patients with mild to moderate Type 1 Gaucher disease, for whom enzyme replacement therapy is not a therapeutic option. — Treatment of progressive neurological manifestations in adult and paediatric patients with Niemann-Pick type C disease.

Dosing overview

It is recommended to take ZAVESCA without food. In patients with renal impairment, dose adjustment is necessary: those with creatinine clearance of 50–70 mL/min/1.73m² should commence at 100 mg twice daily, and those with 30–50 mL/min/1.73m² at one 100 mg capsule per day.

Key safety warnings

Tremor occurs in approximately 38% of patients with Type 1 Gaucher disease and 58% of patients with Niemann-Pick type C disease; in Type 1 Gaucher disease it is described as an exaggerated physiological tremor of the hands that usually begins within the first month and in many cases resolves after 1 to 3 months of continued treatment. Cases of peripheral neuropathy have been confirmed by electrodiagnostic testing in patients treated with ZAVESCA, primarily in those with relevant concurrent conditions such as vitamin B₁₂ deficiency and monoclonal gammopathy; all patients receiving ZAVESCA should undergo formal baseline and repeat neurological evaluation at 6-month intervals. Gastrointestinal events, mainly diarrhoea, occur in more than 80% of patients; the mechanism is probably inhibition of disaccharidases in the gastrointestinal tract, and miglustat-induced gastrointestinal events respond to individualised diet modification, taking ZAVESCA between meals, and/or anti-diarrhoeal medication such as loperamide. Cases of Crohn's disease have been reported post-marketing in Niemann-Pick type C disease patients; in patients with chronic diarrhoea and/or abdominal pain that do not respond to interventions or in the event of clinical worsening, the possibility of Crohn's disease should be considered. Reliable contraceptive methods should be maintained whilst male patients are taking ZAVESCA and for 3 months following discontinuation, as studies in rats have shown that miglustat adversely affects spermatogenesis, sperm parameters and reduces fertility.

Contraindications

Hypersensitivity to the active substance or to any of the excipients.

PBS listing

No information regarding PBS listing, item number, restriction type or ex-manufacturer price is contained in the provided source documents.

Regulatory history

ZAVESCA miglustat 100 mg capsules were first listed on the ARTG on 23 October 2007. The product information was first approved on 10 February 2011. The TGA approved an extension of indication for the treatment of progressive neurological manifestations in adult and paediatric patients with Niemann-Pick disease Type C on 3 February 2010.