How many drugs treat Late-onset Pompe disease?

Drug Landscape tracks 4 marketed treatments for Late-onset Pompe disease (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

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Subscribe to /feed/disease/late-onset-pompe-disease.xml in Feedly / Inoreader. Every regulatory action, PDUFA date, or trial completion in this treatment area lands in your feed automatically.

Last reviewed 2026-06-10 · How we verify

Late-onset Pompe disease — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Late-onset Pompe disease (disease) competitive landscape: 4 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

4 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Late-onset Pompe disease, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	First approval
Pombiliti	CIPAGLUCOSIDASE ALFA	Amicus Therapeutics Europe Limited		Cation-independent mannose-6-phosphate receptor	2023-01-01
Neogaa	AVALGLUCOSIDASE ALFA	Sanofi	Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC]		2021-01-01
NEXVIAZYME	AVALGLUCOSIDASE ALFA-NGPT	GENZYME CORP	Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC]
POMBILITI	CIPAGLUCOSIDASE ALFA-ATGA	AMICUS THERAP US		Cation-independent mannose-6-phosphate receptor

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

AMICUS THERAP US · 1 drug in Late-onset Pompe disease
Amicus Therapeutics Europe Limited · 1 drug in Late-onset Pompe disease
GENZYME CORP · 1 drug in Late-onset Pompe disease
Sanofi · 1 drug in Late-onset Pompe disease

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Every new regulatory action, PDUFA date, or trial completion in Late-onset Pompe disease:

Cite this brief

Drug Landscape (2026). Late-onset Pompe disease — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/late-onset-pompe-disease. Accessed 2026-06-10.

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