Eliglustat is an orally active small-molecule substrate reduction therapy (SRT) used for the treatment of Gaucher disease type 1 in adults who are CYP2D6 extensive, intermediate, or poor metabolizers, subject to applicable prescribing requirements.
Eliglustat is a ceramide analogue that selectively inhibits glucosylceramide synthase, an enzyme involved in the synthesis of glucosylceramide. By reducing the production of glucosylceramide, eliglustat helps decrease the accumulation of glucosylceramide and related glycosphingolipids in patients with Gaucher disease.
Eliglustat has the molecular formula C23H36N2O4 and a molecular weight of approximately 404.54 g/mol. Its CAS Registry Number is 491833-29-5.
Eliglustat API is a pharmaceutical active ingredient used in the development and manufacture of medicines for Gaucher disease type 1. As a substrate reduction therapy, eliglustat works by reducing the synthesis of glucosylceramide rather than directly replacing the deficient lysosomal enzyme.
Eliglustat API is relevant to pharmaceutical formulation development, analytical research, quality-control testing and manufacturing of substrate reduction therapy products.
| 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% |
| Heavy Metals | NMT 20 ppm |
| pH (1% Solution) | 2.0 – 4.0 |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 2.0% |
| Water Content | NMT 1.0% |
| Melting Point | 168°C – 172°C |