Good news for patient associations with cystic fibrosis : the National Medicines Safety Agency (ANSM), announced at the end of the week the establishment of a temporary cohort use authorization system (ATUc) for the drug Orkambi from the Vertex laboratory. This medicine, which is intended for patients with cystic fibrosis aged 12 years and over, “homozygous for the F508del mutation of the CFTR gene” (the main form of cystic fibrosis) is composed of two molecules: lumacaftor and ivacaftor, which correct certain cellular abnormalities linked to cystic fibrosis.
The results of clinical trials available today show a “moderate but significant improvement in respiratory function” as well as beneficial effects on weight gain, the decrease in the number of courses of antibiotics and the quality of life. According to the French cystic fibrosis registry, 1,500 patients are potentially affected by this device.
What is a temporary authorization for use?
In France, the exceptional use of pharmaceutical specialties that do not benefit from a marketing authorization (MA) and that are not the subject of a clinical trial is subject to the prior obtaining of a Temporary Authorization of ‘Use (ATU).
In the case of Orkambi, this is a so-called “cohort” ATU. This type of authorization concerns medicinal products the efficacy and safety of use of which are strongly presumed and which are intended for a group of patients treated and monitored according to criteria defined in a protocol for therapeutic use. The drug manufacturer then undertakes to file an MA application.
Issued by hospital pharmacies only
During the ATUc period, the Orkambi will be delivered by hospital pharmacies on prescription by doctors from the CF Resource and Skills Centers, for a period of four weeks, renewable. Particular attention will be paid to the possible side effects of the drug.
The next step will be to obtain the marketing authorization in France. Then come negotiations on the selling price, before marketing Orkambi in town pharmacies.
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