dm+d

34669011000001108

New Medicines

CerdelgaType 1 and III Gaucher's disease in children aged ≥2 to <18 years

Information

Cerdelga
Licence extension / variation
Sanofi Genzyme
Sanofi Genzyme

Development and Regulatory status

None
Phase III Clinical Trials
None

Category

Glucosylceramide synthase inhibitor
Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide [4]. It is a pan-ethnic disorder with an overall frequency of around 1 in 40,000 to 1 in 50,000 live births (all variants, including types 1, 2 and 3) (Lancet 2008;372:1263)
Type 1 and III Gaucher's disease in children aged ≥2 to <18 years
Oral