Lumacaftor/ivacaftor (Orkambi®) for the treatment of cystic fibrosis

Zorginstituut Nederland carried out a marginal assessment in relation to extending the specific conditions for the medicinal product lumacaftor/ivacaftor (Orkambi®) for the treatment of cystic fibrosis.

Current situation

Since November 2017 lumacaftor/ivacaftor (Orkambi®) film-coated tablets have been included on List 1B and List 2 of the GVS for use in CF patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene.
As of 1 May 2018, the specific condition was extended to include use in patients aged 6 to 11 years.

Zorginstituut's Advice

Lumacaftor/ivacaftor (Orkambi®) has already been placed on List 1B with a specific condition. Based on the new study results (study 115) and the budget impact analysis, we advise the Minister to extend the List 2 conditions of Orkambi® to include use in patients aged 2 to 5 years. Orkambi® 100 mg/125 mg sachet of granules and 150 mg/188 mg sachet of granules can be added to list 1B. This extension in the specific condition will be accompanied by additional costs amounting to €11 million.

Extension in the specific condition of lumacaftor/ivacaftor

Only for cystic fibrosis (CF) patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene.