Pombiliti FDA Approval History
Last updated by Judith Stewart, BPharm on Oct 17, 2023.
FDA Approved: Yes (First approved September 28, 2023)
Brand name: Pombiliti
Generic name: cipaglucosidase alfa-atga
Dosage form: Lyophilized Powder for Injection
Company: Amicus Therapeutics, Inc.
Treatment for: Pompe Disease
Pombiliti (cipaglucosidase alfa-atga) is a hydrolytic lysosomal glycogen-specific enzyme used in combination with Opfolda (miglustat) for the treatment of adults with late-onset Pompe disease.
- Pombiliti is indicated in combination with Opfolda 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.
- Pombiliti is administered as an intravenous infusion over approximately 4 hours, every other week. The infusion is initiated approximately one hour after oral administration of Opfolda.
- Pombiliti carries a Boxed Warning for hypersensitivity reactions (including anaphylaxis), infusion-associated reactions (IARs), and risk of acute cardiorespiratory failure in susceptible patients. Warnings and precautions include embryo-fetal harm.
- Common adverse reactions include headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.
Development timeline for Pombiliti
Further information
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