Taliglucerase Alfa 200U Suppliers & Bulk Manufacturers

Available Forms: Injection

Available Strengths: 200U

Reference Brands: Elelyso (USA/EU)

Category: Orphan Drugs

Taliglucerase alfa is a recombinant form of the human enzyme glucocerebrosidase produced in carrot cells using plant cell-expressed technology. It is used as an enzyme replacement therapy for patients with Type 1 Gaucher disease, a rare genetic disorder caused by deficiency of this enzyme. Taliglucerase alfa works by breaking down accumulated glucocerebroside in cells, helping to reduce liver and spleen enlargement and improve anemia and bone symptoms. It offers a plant-based alternative to other enzyme therapies, providing comparable efficacy and safety.

Product Description:

Taliglucerase alfa is a recombinant glucocerebrosidase, the enzyme missing in Gaucher disease. Its distinguishing feature is how it is made: it is produced in genetically engineered carrot plant cells rather than in mammalian cell culture, and it was the first plant-cell-derived recombinant protein approved for human use anywhere. Plant cells naturally add the terminal mannose residues that macrophages recognise, so the enzyme is taken up by the cells where the stored substrate accumulates without the post-production enzymatic modification that mammalian-derived versions require.

It is used as enzyme replacement therapy for type 1 Gaucher disease, given by intravenous infusion every two weeks. It reduces liver and spleen volume, improves anaemia and platelet counts, and improves bone disease over time. Infusion reactions and hypersensitivity are the main adverse effects, and anti-drug antibodies can develop. It does not treat the neurological forms of Gaucher disease, since the enzyme does not cross the blood-brain barrier.

Sourcing notes. Originator biologic, cold chain, supplied as lyophilised powder for reconstitution. It competes with two other enzyme replacement products and with oral substrate reduction therapy. Its plant-cell platform has been presented as a supply-security argument, which matters to buyers who remember the imiglucerase shortage.