Elosulfase Alfa is a recombinant human lysosomal enzyme and enzyme replacement therapy (ERT) used for the treatment of mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.
Elosulfase Alfa is a recombinant form of N-acetylgalactosamine-6-sulfatase (GALNS). In patients with MPS IVA, deficiency of GALNS results in accumulation of certain glycosaminoglycans, particularly keratan sulfate and chondroitin-6-sulfate. Elosulfase Alfa provides exogenous GALNS enzyme to support the breakdown of these accumulated substrates.
Elosulfase Alfa is a biological pharmaceutical active ingredient and differs from conventional small-molecule APIs because it is a recombinant protein produced using biotechnology.
Elosulfase Alfa is a recombinant enzyme pharmaceutical ingredient relevant to the development and manufacture of enzyme replacement therapies for MPS IVA. As a biologic, its quality evaluation involves protein characterization, biological activity, purity, aggregation, glycosylation and other critical quality attributes in addition to conventional analytical testing.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| pH (1% Solution) | 5.5 – 6.5 |
| Individual Impurity | ≤ 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |
| Melting Point | 150°C – 154°C |