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laronidase (Aldurazyme®)

Reference No. 180

Publication date:

Appraisal information

laronidase (Aldurazyme®) 100 units/ml concentrate for solution for infusion

Company: Genzyme Therapeutics
BNF category: Nutrition and blood
NMG meeting date: Not scheduled
AWMSG meeting date: 01/12/2004
Submission Type: Full Submission
Status: Recommended with restrictions
Advice No: Not available
Ratification by Welsh Government: 28/02/2005

Current Progress

Ratification by
Welsh Government

AWMSG advice

The Minister for Health and Social Services has endorsed AWMSG's recommendation to support the use of laronidase within NHS Wales. It has been agreed that: AWMSG would support the use of laronidase (Aldurazyme®) within NHS Wales subject to the following restrictions:- 1. Use of laronidase will be in accordance with:- (a) the drug’s Summary of Product Characteristics (SPC), subject to paragraph 2 below; and (b) agreed uniform service standards and clinical guidelines; 2. AWMSG recognises that some current uses of laronidase fall outside the drug’s SPC (such as its short term use before and after bone marrow transplantation). Any use which falls outside the SPC will only be supported if part of an approved clinical trial. Patients from Wales will be treated at either:- (a) the specialist centre for the treatment of lysosomal storage disorders at the University Hospital of Wales, Cardiff; or (b) one of the six centres which will be nationally designated and funded by the Department of Health under the auspices of the National Specialised Commissioning Advisory Group to provide a service for patients with lysosomal storage disorders; 3. Having received appropriate consent, details of patients receiving treatment will be entered into the Registry for MPS1 held by the Society for Mucopolysaccharide Diseases (the MPS Society).
Final Appraisal Recommendation (FAR)