Miglustat is water soluble and 70-80% of the dose is excreted unchanged in urine. It is not a substrate, inhibitor, or inducer of CYP enzymes. Risk for gastrointestinal adverse events in the form of nausea, vomiting, diarrhoea, loss of weight and reduced appetite motivates vigilance against insufficient intake of food, especially of carbohydrate.

Butyl hydroxymethyl piperidine

Miglustat is indicated for the treatment of adult patients with type 1 Gaucher disease and Niemann-Pick type C disease. Very common side effects that can be potentially porphyrinogenic through reduction in carbohydrate intake and that also can be confused with an acute porphyria attack are nausea, vomiting, abdominal pain, diarrhoea, obstipation, loss of weight, reduced appetite, depression, insomnia, peripheral neuropathy, paraesthesia and impaired nerve conduction.

Miglustat is not bound to plasma proteins. It is water soluble (Maegawa 2009 and McCormack 2003) and about 70-80% of the dose is excreted unchanged in urine. About 5 % of the dose is excreted as glucuronide. The elimination half-life is 6-7 hours. No drug-drug interactions are observed (DRUID and Interaktionsdatabasen), which indicates that miglustat is not a substrate, inhibitor or inducer of CYP enzymes.