How many drugs treat Pompe disease?

Drug Landscape tracks 2 marketed treatments for 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/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

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/

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

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

Marketed treatment landscape

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

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
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]

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

GENZYME CORP · 1 drug in Pompe disease
Sanofi · 1 drug in Pompe disease

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

Cite this brief

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

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing