Opfolda FDA Approval History
Last updated by Judith Stewart, BPharm on Oct 17, 2023.
FDA Approved: Yes (First approved September 28, 2023)
Brand name: Opfolda
Generic name: miglustat
Dosage form: Capsules
Company: Amicus Therapeutics, Inc.
Treatment for: Pompe Disease
Opfolda (miglustat) is an enzyme stabilizer used in combination with Pombiliti (cipaglucosidase alfa-atga) for the treatment of late-onset Pompe disease.
- Opfolda is indicated, in combination with Pombiliti for the treatment of adult patients with late-onset Pompe disease weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
- Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) is an inherited lysosomal disorder caused by a deficiency of alpha-glucosidase (GAA), which results in muscle damage due to the accumulation of glycogen in the lysosomes of the muscle cells. The infantile form of Pompe disease causes significant impact to heart function, while the late-onset form primarily affects skeletal muscles and muscles that control breathing.
- Pombiliti contains cipaglucosidase alfa-atga, a recombinant human GAA enzyme (rhGAA) that enters the muscle cells where it is converted to its active form to break down glycogen. Opfolda is an enzyme stabilizer designed to reduce inactivation of cipaglucosidase alfa-atga in the blood.
- Opfolda is administered orally every other week, approximately one hour before administration of the Pombiliti infusion.
- Warnings and precautions associated with Opfolda include embryo-fetal harm.
- Common adverse reactions include headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.
Development timeline for Opfolda
Further information
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