Orkambi® Side Effects

Question

Good evening
Do you have information on the side effects of this treatment (Orkambi®) available in France since shortly but already widely distributed in CANADA or the USA?
The side effects are severe and there is little feedback to take a decision.
Best regards

Answer

Hello,

You can have access to a the European Medicines Agency (EMA) detailed documentation by clicking on the following link:

www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/003954/WC500197611.pdf


The adverse drug effects are described in § 4.8, pages 14-16. They are essentially digestive (nausea, vomiting especially in early treatment), hepatobiliary (worsening of liver laboratory abnormalities, liver failure) and respiratory (chest tightness, dyspnea, bronchospasm more frequent in patients with a FEV1 value 40% or lower, and justifying in these cases hospitalization for the monitoring of treatment initiation) disorders.
This is a novel therapeutic:
- whose effectiveness has been proven but is less than that seen with ivacaftor in patients with a gating mutation (G551D);
- whose effectiveness is variable between patients, some being "responders" and other little or no responders;
- whose sometimes serious side effects justify to strictly observe contraindications;
- which has interactions with many drugs or foods which justify to be particularly vigilant before the introduction of a new drug.
This is all the more reason, besides the high cost, which led the "New Therapeutic" group of the Cystic Fibrosis French Society to send to the CF Centers doctors recommendations of checkup to be performed before the initiation of treatment and at regular follow-up.
It is important that you make the point with the CF Center doctor to discuss the validity of this treatment.
Hope this answer can help.
Best wishes
Gilles RAULT, MD, Roscoff CF Center

24.04.2016