Active Ingredient: Miglustat
Miglustat is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
For this indication, competent medicine agencies globally authorize below treatments:
For:
Regimen A: In case that patient weight is ≥ 50 kg, oral, 260 milligrams miglustat, once every 2 weeks.
Regimen B: In case that patient weight is ≥ 40 kg and patient weight is ≤ 50 kg, oral, 195 milligrams miglustat, once every 2 weeks.
Miglustat must be used in combination with cipaglucosidase alfa.
The recommended dose is to be taken orally every other week in adults aged 18 years and older and is based on body weight:
Miglustat 65 mg should be taken approximately 1 hour but no more than 3 hours before the start of the cipaglucosidase alfa infusion.
Figure 1. Dose timeline:
* Miglustat should be taken approximately 1 hour but no more than 3 hours before the start of the cipaglucosidase alfa infusion.
Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease. In case of an insufficient response or intolerable safety risks, discontinuation of miglustat in combination with cipaglucosidase alfa treatment should be considered. Both medicinal products should either be continued or discontinued.
If the miglustat dose is missed, treatment should occur as soon as possible. If it is not taken, do not start the cipaglucosidase alfa infusion. Cipaglucosidase alfa infusion can start 1 hour after miglustat is taken.
Miglustat should be taken on an empty stomach.
Patients should fast 2 hours before and 2 hours after taking miglustat. During this 4-hour fasting period, water, fat-free (skimmed) cow’s milk, and tea or coffee with no cream, sugars, or sweeteners can be consumed. The patient can resume normal eating and drinking 2 hours after taking miglustat.
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