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 36m ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
MYOZYME (alglucosidase alfa) is an enzyme replacement therapy indicated for Pompe disease (glycogen storage disease type II), a rare inherited lysosomal disorder caused by deficiency of the GAA enzyme. The drug provides an exogenous source of acid maltase that is internalized via mannose-6-phosphate receptors and transported to lysosomes, where it cleaves accumulated glycogen and restores metabolic function.
LOE Approaching
As LOE approaches, the commercial team is likely consolidating and shifting focus toward lifecycle management and retention strategies in a high-barrier rare disease market.
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
Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years
Started Oct 2022
6 enrolled
Glycogen Storage Disease Type II Late OnsetPompe Disease (Late-onset)Pompe Disease Late-Onset+2 more
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)
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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.