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"Adopt a project" - Foundation for Cystic Fibrosis Research - € 25,000

'Adopt a project' - Foundation for Cystic Fibrosis Research - € 25,000

The UniCredit Foundation contributes to the study of the safety and efficacy of the Kaftrio® drug administered to patients with advanced lung disease

Cystic fibrosis is the most widespread serious genetic disease in Italy; it affects multiple organs, especially the lungs, and there is currently no definitive cure for the over 2,000 known mutations.


For the most frequent mutation, F508, in the United States the drug Trikafta®, known as Kaftrio® in Europe, was shown to have good efficacy in phase 3 human clinical trials. Following approval by the Food and Drug Administration and the European Medicines Agency, the Italian Medicines Agency is currently assessing the drug and whether to permit reimbursement by the Italian National Health Service. The UniCredit Foundation chose to support the FFC as part of its strategic project entitled,
"Il nuovo farmaco per la mutazione F508 alla prova della vita reale: un primo passo riguarderà la condizione di malattia polmonare avanzata" ("The new drug to treat the F508 mutation under real-life conditions: a first step on the condition of advanced pulmonary disease"). The FFC is sponsoring this study in partnership with the Italian
Cystic Fibrosis Society (SIFC), and it will involve cystic fibrosis centers in Italy and around 200 CF patients. The principal investigator for the study is Dr. Cesare Braggion, the director of the FFC's clinical research department. It is a study that will fill the information gap on the efficacy and safety of Kaftrio® in patients with advanced CF (predicted FEV1 <40%) who are excluded from clinical trial authorization, but take the drug on an expanded access basis (as per Italian Ministerial Decree 9/7/2017) as no other valid treatment alternatives exist for them. Some of them have been added to the transplant waiting list.

 

The Partner - Cystic Fibrosis Research Foundation


The Cystic Fibrosis Research Foundation was founded as a non-profit organization in 1997 with the aim of raising awareness of cystic fibrosis, the most common serious genetic disease in Italy - with an estimated 7,000 CF patients in the country and 100,000 in the world around the world - by promoting advanced research projects to improve the duration and quality of life patients and defeating the disease. Recognized by the Italian Ministry of Education, as a sponsor of scientific research on cystic fibrosis it works through a network of 1,000 researchers and more than 140 delegations and support groups throughout Italy. It also has 10,000 volunteers who raise money for and awareness on the disease. Led by Matteo Marzotto as chair, since 2002 the FFC has invested €32 million in 417 research projects. Following an annual call for proposals, studies are selected based on an evaluation, with contributions from international experts and the foundation's scientific committee in collaboration with the Scientific Board led by Professor Gianni Mastella.