Agalsidase Beta 35 mg Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 35 mg

Reference Brands: Fabrazyme (USA/EU)

Category: Orphan Drugs

Agalsidase beta is a recombinant form of the human enzyme alpha-galactosidase A, used in enzyme replacement therapy for Fabry disease. It helps break down globotriaosylceramide (GL-3), a fatty substance that accumulates in the cells of patients with this inherited lysosomal storage disorder. By reducing GL-3 buildup, Agalsidase beta helps alleviate symptoms such as pain, kidney dysfunction, and cardiovascular complications associated with Fabry disease.

Product Description:

Agalsidase beta is a recombinant human alpha-galactosidase A enzyme designed for intravenous administration and approved for the treatment of Fabry disease, a rare, progressive, multisystemic, and potentially life-threatening lysosomal storage disorder. Fabry disease results from a deficiency of the alpha-galactosidase A enzyme, which leads to the accumulation of glycosphingolipids—particularly globotriaosylceramide (GL-3)—within lysosomes across various tissues. This accumulation is a key driver of the disease’s progression, affecting organs such as the kidneys, heart, and nervous system.

Agalsidase beta provides an exogenous source of alpha-galactosidase A, facilitating the breakdown of accumulated GL-3. Clinical studies have demonstrated that intravenous administration of agalsidase beta effectively clears GL-3 from target cells and prevents its reaccumulation over long-term therapy. In post-approval trials, treatment with agalsidase beta was associated with a reduced risk of major clinical events and improved patient outcomes. The therapy is generally well tolerated and represents a significant advancement in the management of Fabry disease. Agalsidase beta therapy is strongly recommended for patients with confirmed Fabry disease who are appropriate candidates for enzyme replacement therapy.