Orkambi® prescription

Question

Hello
My son aged 30, has a F508del mutation but has not been offered ORKAMBI® yet.
His FEV1 is 27% and he made all pre-transplant tests a year ago.
Are all patients taken into consideration for Orkambi or are there cases where this treatment is not recommended?
Has he to request it from the CF Center doctor or just wait the next appointment to propose getting Orkambi®?
Thank you in advance for your response
Cordially

Answer

Hello,

Only CF patients homozygous for the F508del mutation (say carriers of this mutation in duplicate) are eligible for the combination of lumacaftor and ivacaftor (Orkambi®). The treatment has to be prescribed by the doctor of the CF Center in the frame of a temporary use authorization (ATU) in anticipation of the forthcoming marketing authorization decision that has to be made by the health authorities. There are some drug contraindications including, in particular, the existence of liver damage. There are no contraindications linked to a low FEV1, but the more frequent occurrence of potentially severe side effects (bronchospam) in patients with advanced respiratory disease (FEV1 lower than 40% of the expected value for age and gender) incites a hospital-based surveillance in the hours following the first dose.
If your son is homozygous for the F508del mutation, the treatment decision by Orkambi® has to be discussed with the doctor of his CF Center.

Hope this answer can help.
Best wishes
Gilles RAULT, MD, Roscoff CF Center

27.02.2016