BLAINJECTIONINJECTABLE
Approved
Sep 2023
Lifecycle
Growth
Competitive Pressure
30/100
Clinical Trials
4
Data completeness
3/8 — Basic
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. First approved in 2023.
Drug data last refreshed 14h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
POMBILITI (cipaglucosidase alfa-atga) is an enzyme replacement therapy for Pompe disease (glycogen storage disease type II), a rare inherited lysosomal disorder caused by deficiency of acid alpha-glucosidase (GAA). The drug provides exogenous GAA that degrades accumulated glycogen in lysosomes, with a bis-M6P targeting mechanism that enhances cellular uptake and lysosomal delivery. Miglustat co-administration stabilizes the enzyme in circulation to maximize therapeutic efficacy.
Growth
Early-stage growth phase in rare disease market with moderate competitive intensity; team building likely underway to establish market access and prescriber relationships.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (4)
A Global Prospective Observational Registry of Patients With Pompe Disease
Started Feb 2024
500 enrolled
Pompe Disease
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Started Jul 2023
36 enrolled
Glycogen Storage Disease Type II Infantile Onset
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Started Feb 2020
21 enrolled
Pompe Disease (Late-onset)
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Started Dec 2018
125 enrolled
Pompe Disease (Late-onset)
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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.