Advice
Following a full submission.
Laronidase is not recommended for use within NHS Scotland for the treatment of mucopolysaccharidosis I. Laronidase was approved by the EMEA under exceptional circumstances and has been granted orphan drug status. The evidence of its efficacy is therefore limited. No information is presented in the submission to support the therapy being cost effective and therefore the economic case is not demonstrated.
The licence holder has indicated their decision to resubmit.
Download detailed advice26KB (PDF)
Medicine details
- Medicine name:
- Laronidase (Aldurazyme)
- SMC ID:
- 100/04
- Indication:
- Mucopolysaccharidosis I
- Pharmaceutical company
- Genzyme Therapeutics Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Full
- Status
- Not recommended
- Date advice published
- 12 July 2004