BLAIV (INFUSION)POWDERPriority Review
Approved
May 2010
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
20
Data completeness
6/8 — Strong
Priority Review
LUMIZYME (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 2010.
Drug data last refreshed 22h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
LUMIZYME (alglucosidase alfa) is a recombinant lysosomal enzyme replacement therapy for Glycogen Storage Disease Type II (GSD II, Pompe disease), an inherited disorder caused by deficiency of the acid alpha-glucosidase (GAA) enzyme. The drug provides an exogenous source of GAA that binds to mannose-6-phosphate receptors, is internalized into lysosomes, and cleaves accumulated glycogen to restore metabolic function. It is administered as an IV infusion and represents the standard-of-care enzyme replacement approach for this rare genetic disorder.
$0.019BPart D
LOE ApproachingProduct approaching loss of exclusivity with modest Part D spending ($19M) and limited claim volume (331), indicating a stable but narrow patient base typical of rare disease portfolios.
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…
Pharmacologic Class:
Hydrolytic Lysosomal Glycogen-specific Enzyme
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
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.