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reveglucosidase alfa

Development stage: Discontinued
Lead developer: BioMarin Pharmaceutical
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

Reveglucosidase alfa (BMN 701) is an investigational enzyme replacement therapy (ERT) developed by BioMarin Pharmaceutical for the treatment of Pompe disease, specifically late-onset Pompe disease (LOPD). It is a recombinant human acid alpha-glucosidase (rhGAA) fusion protein that utilizes Glycosylation-Independent Lysosomal Targeting (GILT) technology. By incorporating an insulin-like growth factor 2 (IGF2) peptide tag, the drug is designed to bind with high affinity to the cation-independent mannose-6-phosphate receptor (CI-MPR), thereby enhancing the uptake of the enzyme into the lysosomes of muscle cells compared to conventional ERTs. Although it demonstrated clinical activity in Phase 2 and Phase 3 trials, BioMarin discontinued its development in June 2016, citing the significant additional investment and time required to meet regulatory expectations for a competitive product profile.

Other names: reveglucosidase alfa-BioMarin Pharmaceutical-Pompe diseaseIGF2-tagged recombinant human acid alpha-glucosidaseIGF-2-tagged recombinant human acid alpha-glucosidaseIGF 2-tagged recombinant human acid alpha-glucosidase

Targets

IGF2R (Cation-independent mannose-6-phosphate receptor)

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