Miglustat

What it is used for

YARGESA is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option. Type 1 Gaucher Disease YARGESA is indicated as monotherapy for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g.

due to allergy, hypersensitivity, or poor venous access).

Side effects, and when to stop

The following serious adverse reactions are described below and elsewhere in the labeling:

• Peripheral Neuropathy [ see Warnings and Precautions ]
• Tremor [ see Warnings and Precautions ]
• Diarrhea and weight loss [ see Warnings and Precautions ]
• Reductions in platelet count [ see Warnings and Precautions ]

Who should not take it

None

Other medicines and interactions

While co-administration of miglustat capsules appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase [ see Clinical Pharmacology ].