Eliglustat Capsule 84 mg Suppliers & Bulk Manufacturers

Available Forms: Capsules

Available Strengths: 84 mg

Reference Brands: Cerdelga®(US & EU)

Category: Orphan Drugs

Eliglustat 84 mg capsules, an oral glucosylceramide synthase inhibitor for adults with Gaucher disease type 1, dosed by CYP2D6 genotype. For hospital pharmacies, rare disease centres and licensed importers.

Product Description:

Eliglustat is a substrate reduction therapy for Gaucher disease type 1. It inhibits glucosylceramide synthase, the enzyme that makes glucosylceramide, so less of the lipid is produced for the faulty glucocerebrosidase enzyme to clear. It is an 84 mg hard capsule taken by mouth. For eligible adults this offers an alternative to enzyme replacement therapy given by intravenous infusion every two weeks.

Every patient needs CYP2D6 genotyping first, because the dose depends on how fast they metabolise the drug. Extensive and intermediate metabolisers take 84 mg twice a day, poor metabolisers 84 mg once a day, and ultra-rapid or undetermined metabolisers should not use it. Strong or moderate CYP2D6 and CYP3A inhibitors raise levels, some combinations are contraindicated, and strong CYP3A inducers make it ineffective. Grapefruit products are avoided. High levels can prolong the QT interval, so it is used with care in heart disease and avoided with class IA and III antiarrhythmics. It is not indicated for Gaucher types 2 or 3.

Sourcing notes. Eliglustat is an orphan medicine registered in a limited number of markets, and demand is counted per patient, so most orders are small, regular and tied to a named prescriber. Check that the treating centre can arrange CYP2D6 genotyping. The capsules are stored at room temperature in the original blister, with no cold chain. Ask for the batch CoA, GMP certificate and CoPP, or the special-access import permit where it is unregistered. It is prescription-only. State monthly capsule need, which differs for once- and twice-daily patients.