CF Patients in Italy to Gain Access to Orkambi Under National … – Cystic Fibrosis News Today

The Italian health insurance systemhas agreed to cover cystic fibrosispatients use ofVertex PharmaceuticalsOrkambi (lumacaftor/ivacaftor).

Those eligible for the Italian Medicines Agency coverage are patients 12 and older who have two copies of a particular mutation of the gene that is defective in the disease. That is the F508del mutation of the CF transmembrane conductance regulator, or CFTR, gene.

The Agencia Italiana del FarmacosOrkambi initiative is aimed both atlowering the drugs price and helping more patients obtain access to it. Italys efforts are part of a bigger European initiative. The British and French health insurance systems are continuing to negotiate Orkambi supply and price deals with Vertex.

F508del is a deletion mutation of the CFTR gene, which means that part of the normal gene is missing. The mutation generatesdefective CFTR protein, which leads to inflammation and mucus buildup in the lungs, digestive track, and elsewhere. These problems can cause severe respiratory and digestive dysfunction, as well as other complications like infections.

Two copies of the F508del mutation can result in little-to-no CFTR protein at the cell surface where it is needed.

Orkambi is a combination therapy composed of lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of mature protein at the cell surface, and ivacaftor to enhance the activity of the CFTR protein once it reachesthe surface.

The U.S. Food and Drug Administration approved Orkambifor the treatment of CF in July 2015.

It based its approval on results of two clinical trials, TRAFFIC (NCT01807923) and TRANSPORT (NCT01807949). Those studies showed that the drug,taken as two pills every 12 hours, improved lung function in CF patients, when compared with a placebo. It also reduced pulmonary exacerbation rates.

We are pleased to have reached this agreement on behalf of CF patients inItalywho have been waiting for this important medicine, Simon Bedson, senior vice president of Vertex, said in a press release. We continue negotiations with other countries, includingFranceand theUnited Kingdom, and we encourage these national health authorities and governments to work quickly with us to achieve reimbursement for all patients who may benefit.

Italys countrywide sign-off means that regional health coverage authorities there will begin making Orkambi available to patients who are eligible for it.

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CF Patients in Italy to Gain Access to Orkambi Under National ... - Cystic Fibrosis News Today