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.
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LUMIZYME (alglucosidase alfa) is an intravenous enzyme replacement therapy approved in 2010 for Glycogen Storage Disease Type II (Pompe disease), a rare inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). The drug provides an exogenous source of GAA that binds to mannose-6-phosphate receptors on cell surfaces, allowing internalization into lysosomes where it cleaves accumulated glycogen and restores enzymatic function. As the first and historically dominant treatment for this ultra-rare condition, LUMIZYME represents a foundational therapy in the lysosomal storage disease treatment landscape.
(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…
Hydrolytic Lysosomal Glycogen-specific Enzyme
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Avalglucosidase Alfa Pregnancy Study
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
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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.
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Upgrade to Pro — $25/moLUMIZYME currently has 912 open roles linked across the organization, spanning brand management, medical science liaisons, regional field teams, and patient services roles dedicated to managing the ultra-rare Pompe disease patient base and physician relationships. Success in this space requires deep expertise in rare disease commercialization, patient registry management, genetic counseling coordination, and relationships with the small community of metabolic disease specialists and pediatric neurologists. The impending LOE and biosimilar threat create heightened demand for roles focused on lifecycle management, competitive positioning, and payer strategy during this critical transition period.