Yargesa
- Prescription status not stated
- Digestion, stomach, diabetes and nutrition
- Active substance
- Miglustat
- Made by
- Piramal Critical Care B.V.
- Source
- Official label
Quoted from the official source. Not yet reviewed by Mediclarum — the leaflet in your pack is authoritative.
What it is for
- Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
From the official label · 2026-09-30 · European Medicines Agency
How it works
From the US prescribing label for the same active substance (Miglustat). It describes the substance, not this exact product.
Type 1 Gaucher disease is caused by a functional deficiency of glucocerebrosidase, the enzyme that mediates the degradation of the glycosphingolipid glucosylceramide.
Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids.
In healthy subjects, 67% of the administered dose was excreted unchanged in urine over 72 hours.
Quoted from the prescribing label, sections “Mechanism of Action” and “Pharmacokinetics”. DailyMed · 2025-12-31
Same active substance, strength and form in other countries
Matched on active substance, strength and kind of form only. This is not a statement that the products are equivalent or interchangeable: other ingredients, release and approved uses can differ — ask a pharmacist before swapping.
Medicine passport: one printable page to show a pharmacist abroad
United StatesNo exact match for this strength and form
SpainNo exact match for this strength and form
FranceNo exact match for this strength and form
CanadaNo exact match for this strength and form
NetherlandsNo exact match for this strength and form
SwitzerlandNo exact match for this strength and form
Details
| Made by | Piramal Critical Care B.V. |
|---|---|
| Active substance | Miglustat |
| Used in | Digestion, stomach, diabetes and nutrition |
| Classification | A16AX06 · Digestion, stomach, diabetes and nutrition |
| Generic | Yes — a generic medicine, according to the register |
| Authorised on | 2017-03-22 |
| Last updated by the regulator | 2025-12-19 |
| EMA product number | EMEA/H/C/004016 |
Source: European Medicines Agency — medicine data · 2026-09-30 · not reviewed by a clinician
Source: European Medicines Agency (EMA). Product information as published by EMA. Reuse terms: EMA legal notice (reproduction authorised).
Same active substance
These contain the same substance. That does not mean one can replace another — ask a pharmacist.