OPFOLDA

What it is

OPFOLDA is a medicine containing 65 mg of miglustat per hard capsule. It is a size 2 hard gelatin capsule with a grey opaque cap and a white opaque body, containing white to off-white powder. OPFOLDA is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

Approved indications

— Long-term enzyme replacement therapy in combination with cipaglucosidase alfa for adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

Dosing overview

OPFOLDA must be used in combination with cipaglucosidase alfa. The recommended dose of OPFOLDA taken orally every other week is 4 capsules (260 mg) for patients weighing 50 kg or more, and 3 capsules (195 mg) for patients weighing 40 kg to less than 50 kg. OPFOLDA should be swallowed whole and taken on an empty stomach with water only. The patient must fast 2 hours before and 2 hours after taking OPFOLDA. Cipaglucosidase alfa infusion should start 1 hour after taking OPFOLDA.

Key safety warnings

OPFOLDA has a direct effect on the enzymatic function of major disaccharidases of the intestinal epithelium, and inhibits disaccharidases with alpha-glycosidic linkages including sucrase, maltase, and isomaltase. The strength of potential interactions can immediately interfere with digestive activity of sucrose, maltose, and isomaltose leading to maldigestion, osmotic influx of water, increased fermentation, and production of irritating metabolites. Reliable contraceptive measures must be used by women of childbearing potential during treatment with OPFOLDA in combination with cipaglucosidase alfa, and for 4 weeks after the last dose. There are no clinical data from the use of OPFOLDA in combination with cipaglucosidase alfa in pregnant women, animal studies with miglustat alone and in combination with cipaglucosidase alfa have shown reproductive toxicity, and OPFOLDA in combination with cipaglucosidase alfa therapy is not recommended during pregnancy. Patients should be advised to discontinue breastfeeding while on treatment with OPFOLDA in combination with cipaglucosidase alfa. The most common adverse reactions reported in patients treated with OPFOLDA in combination with cipaglucosidase alfa in all clinical trials were headache, diarrhoea, fatigue, nausea, abdominal pain, pyrexia, and chills. Reported serious adverse reactions in all trials were urticaria, anaphylaxis, chills, cough, flushing, pyrexia, presyncope, dyspnoea, pharyngeal oedema, wheezing, and hypotension.

Contraindications

OPFOLDA is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients. OPFOLDA is contraindicated in patients with contraindication to cipaglucosidase alfa.

Regulatory history

OPFOLDA miglustat 65 mg capsule was first listed on the ARTG on 16 December 2024. The AusPAR approval date was 10 February 2025.