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Drugs / Cipaglucosidase alfa
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Cipaglucosidase alfa

also known as ATB200 · AT GAA · cipa · Cipaglucosidase alfa-atga · Pombiliti

Protein / enzyme biologic

Developed for
glycogen storage disease due to acid maltase deficiency, late-onset · glycogen storage disease II · glycogen storage disease due to acid maltase deficiency, infantile onset
Investigated by
Amicus Therapeutics

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Pombiliti — 2023-03-20 europa.eu ↗

Trials 6

today
EU approved 2023
NCT04808505 · Ph 3 NCT03911505 · Ph 3 NCT04138277 · Ph 3 NCT03729362 · Ph 3 NCT02675465 · Ph 1/2
201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/21 trial
Phase 1/2 NCT02675465 Apr 2016 → Aug 2024 glycogen storage disease II Amicus Therapeutics Completed No outcome recorded
Phase 34 trials · 1 missed
Phase 3 NCT04808505 Jul 2023 → Jul 2027 expected glycogen storage disease due to acid maltase deficiency, infantile onset Amicus Therapeutics Recruiting No outcome recorded
Phase 3 NCT03911505 Feb 2020 → Mar 2027 expected glycogen storage disease due to acid maltase deficiency, late-onset Amicus Therapeutics Active not recruiting No outcome recorded
Phase 3 NCT04138277 Dec 2019 → Dec 2024 glycogen storage disease due to acid maltase deficiency, late-onset Amicus Therapeutics Completed No outcome recorded
Phase 3 NCT03729362 Dec 2018 → Dec 2020 glycogen storage disease due to acid maltase deficiency, late-onset Amicus Therapeutics Completed Missed primary
Phase not stated1 trial
— NCT03865836 — glycogen storage disease II Amicus Therapeutics Available No outcome recorded
Comparator or background therapy1 trial
— NCT04327973 Background — glycogen storage disease due to acid maltase deficiency, infantile onset Amicus Therapeutics Available No outcome recorded

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2026-10-02 BioMarin Pharmaceutical Results BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress biomarin.com ↗
These results from the PROPEL open-label extension (OLE) study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up.

All press releases naming this drug 1 release

DateIssuerRelease

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 9 sources stand behind the page.

FieldValueCited text
Known as Cipaglucosidase alfa ClinicalTrials.gov intervention name — accepted as the source's own label NCT04808505 ↗
2

NCT03911505 ↗

NCT03729362 ↗
Known as AT GAA ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗
Known as ATB200 ClinicalTrials.gov intervention name — accepted as the source's own label NCT04138277 ↗
3

NCT04327973 ↗

NCT02675465 ↗

NCT03865836 ↗
Known as cipa “Cipaglucosidase alfa (cipa) is an enzyme replacement therapy that aims to replace the missing enzyme.” PMID 41631150 ↗ Jan 2026
Known as Cipaglucosidase alfa-atga ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗
Known as Pombiliti ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗
Action Restore “Cipaglucosidase alfa (cipa) is an enzyme replacement therapy that aims to replace the missing enzyme.” PMID 41631150 ↗ Jan 2026
Modality Protein / enzyme biologic “a highly targeted rhGAA (ATB200)” NCT02675465 ↗
Route Intravenous “Sequential single ascending doses of intravenously infused ATB200” NCT02675465 ↗