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alglucosidase alfa (Myozyme™)

Reference No. 17

Publication date:

Appraisal information

alglucosidase alfa (Myozyme™) 50 mg powder for concentrate for solution for infusion

Company: Genzyme Therapeutics
BNF category: Nutrition and blood
NMG meeting date: Not scheduled
AWMSG meeting date: 02/10/2006
Submission Type: Full Submission
Status: Recommended with restrictions
Advice No: Not available
Ratification by Welsh Government: 25/10/2006

Current Progress

Ratification by
Welsh Government

AWMSG advice

It was agreed (by majority) that the recommendation to the Minister for Health & Social Services with regard to alglucosidase alfa (Myozyme™) is that alglucosidase alfa (Myozyme™) should be endorsed within NHS Wales for the treatment of Pompe disease in accordance with the licensed indication but with the specific restriction. That there is presently insufficient evidence of clinical effectiveness in late-onset disease and AWMSG does not endorse its use in this group of patients at this stage. AWMSG endorsed the use of alglucosidase alfa (Myozyme™), but with the specific exclusion of alglucosidase alfa (Myozyme™) in Late-onset Pompe disease of the Adult Onset form (Adult Onset disease) because of insufficient evidence of clinical effectiveness. The Committee was however persuaded that alglucosidase alfa (Myozyme™) should be available for the treatment of Late Onset Pompe disease of the Juvenile Onset form (Juvenile Onset Disease). AWMSG recommends that: 1. Patients receiving alglucosidase alfa (Myozyme™) will be entered into the Pompe registry. 2. Treatment will be administered under the supervision of a physician experienced in the management of Pompe disease or other neuromuscular disorders. 3. Treatment will be administered according to agreed guidelines at appropriate centres. 4. The AWMSG recommendation will be reviewed in light of further evidence becoming available.
Final Appraisal Recommendation (FAR)