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Last reviewed 2026-06-10 · How we verify

Lysosomal alpha-1,4-glucosidase deficiency - infantile onset — Treatment Landscape & Competitive Intelligence

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

Lysosomal alpha-1,4-glucosidase deficiency - infantile onset (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 Lysosomal alpha-1,4-glucosidase deficiency - infantile onset, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
Rhgaa	ALGLUCOSIDASE ALFA	Sanofi	Hydrolytic Lysosomal Glycogen-specific Enzyme [EPC]			2006-01-01
Myozyme	Myozyme	Genzyme, a Sanofi Company

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, a Sanofi Company · 1 drug in Lysosomal alpha-1,4-glucosidase deficiency - infantile onset
Sanofi · 1 drug in Lysosomal alpha-1,4-glucosidase deficiency - infantile onset

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Every new regulatory action, PDUFA date, or trial completion in Lysosomal alpha-1,4-glucosidase deficiency - infantile onset:

Cite this brief

Drug Landscape (2026). Lysosomal alpha-1,4-glucosidase deficiency - infantile onset — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/lysosomal-alpha-1-4-glucosidase-deficiency-infantile-onset. Accessed 2026-06-10.

Lysosomal alpha-1,4-glucosidase deficiency - infantile onset full disease profile — treatment pathway, diagnostics, guidelines
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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing