BLAIV (INFUSION)INJECTABLEPriority Review
Approved
Apr 2006
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
20
Data completeness
3/8 — Basic
Priority Review
MYOZYME (alglucosidase alfa) by Sanofi is (acid maltase deficiency, glycogen storage disease type ii, gsd ii, glycogenosis type ii) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme gaa. First approved in 2006.
Drug data last refreshed 1h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
MYOZYME (alglucosidase alfa) is an intravenous enzyme replacement therapy for acid maltase deficiency, also known as Glycogen Storage Disease Type II (GSD II), a rare inherited lysosomal disorder. The drug provides an exogenous source of the deficient GAA enzyme, which is internalized via mannose-6-phosphate receptor binding and transported to lysosomes where it cleaves accumulated glycogen. This mechanism restores enzymatic activity and halts progressive muscle weakness and organ dysfunction in affected patients.
LOE Approaching
Product is nearing loss of exclusivity with moderate competitive pressure (30/100), signaling transition planning and potential team restructuring.
Mechanism of Action
(acid maltase deficiency, glycogen storage disease type II, GSD II, glycogenosis type II) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of the lysosomal enzyme GAA. Alglucosidase alfa provides an exogenous source of GAA. Binding to mannose-6-phosphate…
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Started May 2025
13 enrolled
Glycogen Storage Disease Type IIPompe's Disease
Avalglucosidase Alfa Pregnancy Study
Started Oct 2022
100 enrolled
Pompe DiseasePregnancy
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Started Jul 2022
17 enrolled
Glycogen Storage Disease Type II
A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Started Oct 2021
57 enrolled
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Started Sep 2021
17 enrolled
Glycogen Storage Disease Type II
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.