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Drug

Cipaglucosidase alfa

Approved medicine
ResearchOngoing clinical research

Also known as Pombiliti, Recombinant human acid alpha-glucosidase optimized for high expression of mannose 6-phosphate.

RxNorm2667433ChEMBLCHEMBL4594329ATCA16AB23UNII4SED7F4BSG
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Clinical trial2026-06-01Primary completion · ClinicalTrials.gov

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

Clinical trial2026-01-22Results expected Q3 2027 · ClinicalTrials.gov

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

Clinical trial2025-09-11Results posted · ClinicalTrials.gov

Approval: Pombiliti (EMA)

Regulatory2023-03-20EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Cipaglucosidase alfa
Aliases & brands
PombilitiRecombinant human acid alpha-glucosidase optimized for high expression of mannose 6-phosphate
RxNorm CUI
2667433
ChEMBL ID
CHEMBL4594329
ATC codes
A16AB23
UNII
4SED7F4BSG
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2023-03-20
Latest approval
2023-03-20
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2023-03-20Approval
Approval: Pombiliti (EMA)
Indication: Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset… Show full indication

Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

3 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
3
registered trials across all phases
LATEST COMPLETION 2021
2
Active studies
1
Recruiting
3
Late-stage (III+)
1
Completed
PHASE DISTRIBUTIONn = 3
Phase 33

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

RecruitingPH IIIAmicus Therapeuticsn=362027

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Active, not recruitingPH IIIAmicus Therapeuticsn=212026

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

CompletedPH IIIAmicus Therapeuticsn=1252021
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.