BLAINJECTIONINJECTABLE
Approved
Sep 2023
Lifecycle
Growth
Competitive Pressure
30/100
Clinical Trials
4
Data completeness
4/8 — Partial
POMBILITI (cipaglucosidase alfa-atga) by Amicus Therapeutics is (also known as glycogen storage disease type ii, acid maltase deficiency, and glycogenosis type ii) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (gaa) that degrades glycogen to glucose in the lysosome. Approved for pompe disease, glycogen storage disease type ii. First approved in 2023.
Drug data last refreshed 20h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
POMBILITI (cipaglucosidase alfa-atga) is a recombinant lysosomal enzyme replacement therapy for Pompe disease (glycogen storage disease type II), a rare inherited disorder caused by deficiency of acid alpha-glucosidase (GAA). The drug provides exogenous GAA that degrades accumulated glycogen in lysosomes, with a unique bis-M6P targeting mechanism that enables high-affinity cellular uptake and lysosomal delivery; miglustat co-administration stabilizes the enzyme in circulation.
Growth
Recently approved biologic in growth phase; early commercial expansion underway with substantial investment in field infrastructure and patient support programs.
Mechanism of Action
(also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in…
Indications (2)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.