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New effective treatment for some Cystic Fibrosis mutations!

Great news for people living with CF! On March 29th, Vertex released two new Phase 3 studies on the use of a CF treatment. Indeed, the association of ivacaftor (activator, active principle of Kalydeco®) and of the corrector VX-661 (tezacaftor) proved effective for patients aged 12 years and older and whose mutation is DF508 combined with one of the 27 other so-called residual mutations: 2789+5G->A, D110E, R352Q, A1067T, 3849+10kbC->T, D110H, A455E, R1070Q, 3272-26A->G, R117C, D579G, R1070W, 711+3A->G, E193K, S945L, F1074L, E56K, L206W, S977F, D1152H, P67L, P205S, F1052V, D1270N, R74W, R347H, K1060T.

Both studies, conducted in Europe and the United States on several hundred patients, demonstrated an average improvement in FEV 1 between 4 and 6.8%. In addition, an improvement in the quality of life in general was observed. Note that Phase 3 of a study is the most advanced in the clinical development of a drug.

At the moment, two treatments are available in Quebec and Canada, Kalydeco and Orkambi. In the case of Orkambi, only people who are homozygous DF508 react to treatment. These new data thus demonstrate that the combination of different potentiators (or activators) makes it possible to find new effective treatments. It is not yet a miracle cure, but we are moving faster than ever towards treatments that will effectively control cystic fibrosis.

According to Jeffrey Chodakewitz, Vertex’s chief medical officer, “this combination of treatment can provide a promising new option for treating the underlying cause of Cystic Fibrosis in the future and bringing us closer and closer to our to develop new drugs for all people with the disease. ”

A request for treatment approval will be made shortly in Europe. Hopefully it will be available quickly here!

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