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Opfolda is a brand name for Miglustat. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: Opfolda (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).
Verbatim from this product's EMA label. Tap a section to expand.
Treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases. Miglustat 65 mg hard capsules must be used in combination with cipaglucosidase alfa.
The summary of product characteristics (SmPC) for cipaglucosidase alfa should be consulted before taking miglustat. Posology The recommended dose is to be taken orally every other week in adults aged 18 years and older and is based on body weight: For patients weighing ≥ 50 kg, the recommended dose is 260 mg (4 capsules of 65 mg).
For patients weighing ≥ 40 kg to < 50 kg, the recommended dose is 195 mg (3 capsules of 65 mg). Miglustat 65 mg hard capsules should be taken approximately 1 hour but no more than 3 hours before the start of the cipaglucosidase alfa infusion.
3 Figure 1. Dose timeline * Miglustat 65 mg hard capsules 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 65 mg hard capsules in combination with cipaglucosidase alfa treatment should be considered. Both medicinal products should either be continued or discontinued.
Missed dose 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. Special populations Renal and hepatic impairment The safety and efficacy of miglustat in combination with cipaglucosidase alfa therapy have not been evaluated in patients with renal and/or hepatic impairment.
When administering every other week, increased plasma miglustat exposure as a result of moderate or severe renal or hepatic impairment is not expected to appreciably impact cipaglucosidase alfa exposures and is not anticipated to affect efficacy and safety of cipaglucosidase alfa in a clinically meaningful manner.
No dose adjustment is required in patients with renal or hepatic impairment. Elderly There is limited experience with the use of miglustat in combination with cipaglucosidase alfa therapy in patients above the age of 65 years old. There is no dose adjustment required in elderly patients.
3%). Tabulated list of adverse reactions The assessment of adverse reactions was informed by subjects treated with miglustat in combination with cipaglucosidase alfa therapy from the pooled safety analysis across the 3 clinical trials.
0 months. Adverse reactions are listed by MedDRA system organ class in Table 1. The corresponding frequency categories are defined as follows: very common (≥ 1/10), common (≥ 1/100 to < 1/10), uncommon (≥ 1/1 000 to < 1/100), rare (≥ 1/10 000 to < 1/1 000), very rare (< 1/10 000), and not known (cannot be estimated from available data).
Table 1:
Summary of adverse reactions of miglustat-treated patients System organ class (SOC) Frequency Adverse reaction (preferred term) Immune system disorders Common Anaphylactic reaction7 Uncommon Hypersensitivity Nervous system disorders Very common Headache Common Tremor, dysgeusia, paraesthesia Uncommon Balance disorder, migraine4 Cardiac disorders Common Tachycardia6 Vascular disorders Common Hypotension Uncommon Pallor Respiratory, thoracic and mediastinal disorders Common Dyspnoea Uncommon Asthma Gastrointestinal disorders Common Diarrhoea, nausea, abdominal pain1, flatulence, abdominal distension, vomiting, constipation† Uncommon Abdominal discomfort†, oesophageal spasm, oral pain Skin and subcutaneous tissue disorder Common Urticaria3, rash2, pruritus, hyperhidrosis Uncommon Skin discolouration Musculoskeletal and connective tissue disorders Common Muscle spasms, myalgia, arthralgia, muscular weakness 6 † Reported with miglustat only 1 Abdominal pain, abdominal pain upper, and abdominal pain lower are grouped under abdominal pain.
2 Rash and rash erythematous are grouped under rash. 3 Urticaria, urticaria rash, and mechanical urticaria are grouped under urticaria. 4 Migraine and migraine with aura are grouped under migraine. 5 Hypertension and blood pressure increased are grouped under blood pressure increased.
8).
1. Contraindication to cipaglucosidase alfa.
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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Paediatric population The safety and efficacy of miglustat in combination with cipaglucosidase alfa therapy in paediatric patients less than 18 years old have not yet been established. No data are available. Method of administration Miglustat is for oral use.
Miglustat hard capsule has a crimp to prevent opening the capsule shells and should be swallowed whole and taken on an empty stomach. 2). During this 4-hour fasting period, water, fat-free (skimmed) cow’s milk, and tea or coffee * 4 with no cream, sugars, or sweeteners can be consumed.
The patient can resume normal eating and drinking 2 hours after taking miglustat.
6 Tachycardia and sinus tachycardia are grouped under tachycardia. 7 Anaphylaxis, anaphylactic reaction, and anaphylactoid reaction are grouped under anaphylactic reaction. Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important.
It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.