Lumasiran is a synthetic double-stranded small interfering RNA (siRNA) therapeutic designed to target hydroxyacid oxidase 1 (HAO1) messenger RNA. It is conjugated with an N-acetylgalactosamine (GalNAc) ligand to support targeted delivery to liver cells.
Lumasiran is supplied as lumasiran sodium and has CAS Number 1834610-13-7. Its molecular formula is C530H669F10N173O320P43S6Na43, with an approximate molecular weight of 17,286 Da.
Lumasiran is an RNA interference-based active pharmaceutical ingredient used in the development of treatments for primary hyperoxaluria type 1 (PH1). It works by reducing the production of glycolate oxidase through silencing of HAO1 messenger RNA in hepatocytes.
The molecule contains chemically modified nucleotides and a GalNAc targeting ligand. This design helps facilitate uptake by hepatocytes and supports the RNA interference mechanism.
Lumasiran targets HAO1 messenger RNA in liver cells through the RNA interference pathway. HAO1 produces glycolate oxidase, an enzyme involved in the metabolic pathway leading to oxalate production. By reducing HAO1 expression, lumasiran decreases glycolate oxidase levels and helps reduce oxalate production.
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Lumasiran has CAS Number 1834610-13-7 and is supplied pharmaceutically as lumasiran sodium. The molecular formula is C530H669F10N173O320P43S6Na43, with an approximate molecular weight of 17,286 Da.
Lumasiran is an approved RNA interference therapeutic marketed as OXLUMO. It is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in pediatric and adult patients. It received FDA approval in November 2020.
| 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 1.0% |
| Water Content | NMT 1.0% |