Section 16 · Monograph
Cipaglucosidase alfa + miglustat
Reviewed Nov 2026FDA label
Pombiliti + OpfoldaEnzyme replacement + enzyme stabilizer
- Forms
- IV: 105mg vial (Pombiliti) | Cap: 65mg (Opfolda)
- Dose
- Late-onset Pompe, adults 40kg+: 20mg/kg IV every 2 weeks (about 4h) with Opfolda 260mg (50kg+) or 195mg (40 to <50kg) orally
- MOA
- Replaces GAA; miglustat stabilizes the enzyme in blood
Black Box Warning
- Pombiliti: severe hypersensitivity reactions including anaphylaxis, infusion-associated reactions, and risk of acute cardiorespiratory failure in susceptible patients — appropriate medical support must be available during infusion
Indications
- Late-onset Pompe disease in adults weighing 40kg or more who are not improving on their current enzyme replacement therapy (Pombiliti used in combination with Opfolda)FDA-labeled
Mechanism of Action
- Cipaglucosidase alfa is a recombinant GAA with high mannose-6-phosphate content; miglustat stabilizes the enzyme in the bloodstream, increasing delivery to muscle and lysosomal glycogen clearance
Full Dosing
Adult / usual
- Pombiliti 20mg/kg IV every 2 weeks over about 4 hours, given in combination with Opfolda
- Opfolda 260mg (4 capsules) for weight 50kg or more, or 195mg (3 capsules) for 40 to <50kg, taken orally about 1 hour before the infusion with fasting before and after per label
- Start two weeks after the last dose of prior ERT; do not combine with Lumizyme or Nexviazyme
Contraindications
- Embryo-fetal toxicity concern: not recommended in pregnancy — verify the current contraindication status in the prescribing information
- Pombiliti must not be given if Opfolda was not taken as directed
Drug Interactions
- Avoid with other miglustat-containing products
- Premedicate with antihistamines, antipyretics and/or corticosteroids as indicated
Adverse Effects
- Infusion-associated reactions
- Anaphylaxis and hypersensitivity
- Acute cardiorespiratory failure risk
- Headache, diarrhea, fatigue, nausea, abdominal pain (more common with miglustat)
Monitoring Parameters
- Close monitoring during infusion
- Cardiorespiratory status
- Pregnancy status and contraception counseling
Pregnancy & Lactation
- Embryo-fetal toxicity; use effective contraception during and after treatment
- Lactation: weigh benefit and risk; consult current label