Basic Information
A16AX06
miglustat
Other alimentary tract and metabolism products
Therapeutic indication
Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).
Overview Summary
Opfolda is a medicine used in the treatment of adults with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency), an inherited disorder in which patients have breathing difficulties and muscle weakness. It is used in combination with another medicine, cipaglucosidase alfa.
Opfolda is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but it is available as capsules of a different strength.The reference medicine for Opfolda is Zavesca.
Opfolda contains the active substance miglustat.
Active Substances (2)
miglustat
miglustat
Documents (9)
Opfolda : EPAR - Public Assessment Report
July 7, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Opfolda
April 26, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
Opfolda : EPAR - Medicine Overview
July 7, 2023
OVERVIEW_DOCUMENT
Opfolda : EPAR - Public Assessment Report
July 7, 2023
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
CHMP summary of positive opinion for Opfolda
April 26, 2023
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Opfolda : EPAR - Risk management plan summary
July 7, 2023
RISK_MANAGEMENT_PLAN_SUMMARY
Opfolda : EPAR - Product Information
July 7, 2023
DRUG_PRODUCT_INFORMATION
Opfolda : EPAR - All authorised presentations
July 7, 2023
AUTHORISED_PRESENTATIONS
Opfolda: EPAR - Procedural steps taken and scientific information after authorisation
September 22, 2023
CHANGES_SINCE_INITIAL_AUTHORISATION
Overview Q&A (7)
Question
What are the risks associated with Opfolda?
Answer
For the full list of side effects and restrictions with Opfolda, see the package leaflet.
The most common side effect with Opfolda (which may affect up to 1 in 10 people) is constipation.
Opfolda must not be used by people who cannot take cipaglucosidase alfa.
Question
How is Opfolda used?
Answer
Opfolda capsules are taken by mouth approximately 1 hour but no more than 3 hours before a cipaglucosidase alfa infusion (drip).
The medicine can only be obtained with a prescription and treatment should be supervised by a doctor experienced in the management of patients with Pompe disease or similar diseases.
For more information about using Opfolda, see the package leaflet or contact your doctor or pharmacist
Question
How does Opfolda work?
Answer
The active substance in Opfolda, miglustat, attaches to cipaglucosidase alfa during treatment. This makes cipaglucosidase alfa more stable, so it can continue to be absorbed from the blood by the muscle cells that are affected by Pompe disease.
Question
What benefits of Opfolda have been shown in studies?
Answer
One main study in 125 adults with Pompe disease compared the effects of Opfolda plus cipaglucosidase alfa with alglucosidase alfa (another medicine for Pompe disease) plus placebo (a dummy treatment).
The study showed that, after one year of treatment, the distance patients could walk in six minutes improved by around 20 meters in patients treated with Opfolda plus cipaglucosidase alfa, compared with an improvement of around 8 meters in patients on alglucosidase alfa and placebo. This difference was considered clinically relevant.
Question
Why is Opfolda authorised in the EU?
Answer
The European Medicines Agency concluded that the effects of Opfolda in combination with cipaglucosidase alfa are clinically relevant and that their use offers an alternative treatment option for adult patients with late-onset Pompe disease. The safety profile of the combination is considered acceptable and comparable to that of alglucosidase alfa.
The Agency therefore decided that Opfolda’s benefits are greater than its risks and it can be authorised for use in the EU.
Question
What measures are being taken to ensure the safe and effective use of Opfolda?
Answer
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Opfolda have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Opfolda are continuously monitored. Suspected side effects reported with Opfolda are carefully evaluated and any necessary action taken to protect patients.
Question
Other information about Opfolda
Answer
Opfolda received a marketing authorisation valid throughout the EU on 26 June 2023.