Drug intelligence / Profile preview

velaglucerase alfa + vitamin D

Development stage
Unknown
Lead developer
Takeda
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Intravenous, Oral
01

Overview

**Velaglucerase alfa + vitamin D** is a combination of two agents: velaglucerase alfa, a recombinant beta-glucocerebrosidase enzyme replacement therapy, and vitamin D, a secosteroid hormone essential for calcium and bone metabolism. Velaglucerase alfa is produced using gene activation technology in human fibroblast cell lines and is primarily indicated as long-term enzyme replacement therapy for patients with type 1 Gaucher disease. It restores deficient beta-glucocerebrosidase activity, catalyzing the hydrolysis of glucocerebroside within lysosomes, thereby reducing pathological lipid accumulation in liver, spleen, bone, and hematopoietic tissues. Vitamin D (typically cholecalciferol or ergocalciferol) regulates calcium and phosphate homeostasis, enhancing bone mineralization. Combination therapy of velaglucerase alfa and vitamin D would theoretically address the underlying pathophysiology of Gaucher disease while supporting bone metabolism, although no evidence suggests a fixed-dose combination is commercially marketed or clinically validated[1][2][3][4]. The combination represents a rational multi-agent regimen rather than a uniquely branded pharmaceutical product.

Other names
velaglucerase alfavprivvitamin Dergocalciferolcholecalciferol
02

Targets

GBA1 (Acid beta-glucosidase)

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