Italy approves Orkambi for patients with Cystic Fibrosis



Italy has followed Ireland and other EU countries such as Germany, Austria, Denmark and Luxembourg in approving Orkambi for patients with CF who have the particular genotype that stand to benefit from this important drug therapy.

CF Ireland hopes the efforts of the CF Trust in the U.K. will soon result in Orkambi being reassessed positively by regulatory authorities in London, Edinburgh and Belfast. There are many patients in Northern Ireland who could benefit from this drug and we offer our mutual support to them in particular at this time of uncertainty. 

 

Statement from Vertex Pharmaceuticals

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Italian Medicines Agency (Agenzia Italiana del Farmaco, or AIFA) has agreed to reimburse ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The agreement is published online in the Italian Official Gazette. Regional authorities will now begin implementation to provide the hundreds of eligible patients in Italy access to this important medicine. Recent European pricing and reimbursement agreements have enabled broad access to ORKAMBI for thousands of eligible patients in Austria, Denmark, Germany, Ireland, Italy and Luxembourg. Negotiations continue in a number of other countries where CF is prevalent, including France and the United Kingdom.

"We are pleased to have reached this agreement on behalf of CF patients in Italy who have been waiting for this important medicine," said Simon Bedson, Senior Vice President and International General Manager at Vertex. "We continue negotiations with other countries including France and the United Kingdom, and we encourage these national health authorities and governments to work quickly with us to achieve reimbursement for all patients who may benefit."

About ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is available as tablets and is typically taken twice per day.

 

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu