Taliglucerase alfa is a recombinant lysosomal enzyme and a form of human β-glucocerebrosidase (glucocerebrosidase) used as enzyme replacement therapy for Gaucher disease. It is produced using recombinant DNA technology in genetically engineered carrot cell culture. Taliglucerase alfa is a glycosylated monomeric protein with four N-linked glycosylation sites and an approximate molecular mass of 60.8 kDa.
Taliglucerase alfa is marketed as ELELYSO® and is supplied as a sterile, preservative-free, lyophilized product for reconstitution and intravenous infusion. The FDA-approved presentation contains 200 units per single-dose vial.
Swapnroop provides pharmaceutical and biopharmaceutical API solutions for customers requiring enzyme-based biological products, subject to applicable specifications, manufacturing capabilities and regulatory requirements.
Taliglucerase alfa is a recombinant form of human glucocerebrosidase. The enzyme catalyzes the hydrolysis of glucocerebroside into glucose and ceramide. Deficiency of glucocerebrosidase causes glucocerebroside accumulation in lysosomes, particularly within macrophages, which is characteristic of Gaucher disease.
The recombinant enzyme is produced in genetically engineered carrot plant-cell culture, making Taliglucerase alfa notable among recombinant therapeutic proteins.
Unlike conventional chemical APIs, Taliglucerase alfa is a complex recombinant glycoprotein. Its pharmaceutical identity depends on multiple molecular characteristics including amino-acid sequence, glycosylation, higher-order structure, purity and enzymatic activity.
The FDA describes Taliglucerase alfa as a monomeric glycoprotein containing four N-linked glycosylation sites, with mannose-terminated oligosaccharides. These glycan structures contribute to uptake of the enzyme by macrophages.
A database record gives the protein formula as C2580H3918N680O727S17 and molecular weight around 56.64 kDa, illustrating why database molecular-weight values can differ from the approximately 60.8 kDa reported for the glycosylated pharmaceutical protein. For a commercial CMS, the manufacturer's validated specification should take precedence.
Taliglucerase alfa is used as enzyme replacement therapy for Gaucher disease, a lysosomal storage disorder associated with deficient glucocerebrosidase activity.
Pharmaceutical applications include:
The FDA-approved product is administered by intravenous infusion.
As a recombinant enzyme, Taliglucerase alfa requires biological-product characterization rather than conventional small-molecule testing.
Relevant quality attributes can include:
The enzymatic activity is particularly important. The FDA defines one unit as the amount of enzyme that catalyzes hydrolysis of 1 micromole of the synthetic substrate p-nitrophenyl-β-D-glucopyranoside per minute at 37°C.
Swapnroop supports pharmaceutical and biopharmaceutical requirements involving specialized biological products and enzyme-based APIs.
Taliglucerase alfa requirements can be evaluated for:
For Taliglucerase alfa requirements, customers can contact Swapnroop for current specifications, availability, documentation and commercial information.
Taliglucerase alfa is supplied as a sterile, preservative-free, lyophilized powder requiring reconstitution before administration. The approved product is packaged as a 200-unit single-dose vial.
The approved product is stored under refrigerated conditions and requires controlled handling appropriate for a biological product. Exact storage requirements should always be taken from the applicable manufacturer's specification and stability data.
Depending on applicable requirements, documentation may include:
Swapnroop supports pharmaceutical and biopharmaceutical requirements for specialized biological products. Taliglucerase alfa is a complex recombinant enzyme requiring appropriate biological characterization, potency testing and quality-control procedures.
For Taliglucerase Alfa API, biological drug-substance requirements, technical specifications and bulk enquiries, contact Swapnroop.
| Parameter | Specification |
|---|---|
| Appearance | white to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | NLT 90.0% |
| Loss on Drying | NMT 5.0% |
| pH (1% Solution) | 5.5–6.5 |
| Individual Impurity | NMT 1.0% |
| Total Impurities | NMT 5.0% |
| Water Content | NMT 5.0% |