Drug intelligence / Profile preview

alglucosidase alfa + methotrexate + rituximab

Development stage
Unknown
Lead developer
Sanofi
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intravenous, Oral (methotrexate)
01

Overview

This is a **combination regimen** comprising three distinct drugs: - **Alglucosidase alfa** is a recombinant human acid alpha-glucosidase enzyme indicated for enzyme replacement therapy in **Pompe disease (acid alpha-glucosidase deficiency)**. It works by replacing the deficient enzyme responsible for glycogen degradation in lysosomes[1][4][5][6]. - **Methotrexate** is a folate analog **antimetabolite and immunosuppressant** that inhibits dihydrofolate reductase, leading to impaired DNA synthesis, and is primarily used in oncology, autoimmune diseases (such as rheumatoid arthritis), and as an immunosuppressant. - **Rituximab** is a **CD20-directed cytolytic monoclonal antibody** used as an immunotherapy/targeted therapy for B-cell malignancies (e.g., non-Hodgkin's lymphoma, chronic lymphocytic leukemia) and autoimmune diseases (most notably in combination with methotrexate for rheumatoid arthritis)[3]. There is no evidence of this three-drug combination existing as a single marketed product, fixed-dose combination, or uniquely branded regimen. The components may be used together off-label in rare clinical scenarios, but their primary indications and mechanisms are distinct and not overlapping in an approved product format.

02

Targets

DHFR (Dihydrofolate reductase)GAA (Lysosomal acid alpha-glucosidase)CD20 (B-lymphocyte antigen CD20)IGF2R (Cation-independent mannose-6-phosphate receptor)

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