As we informed you in early July, the US Food and Drug Administration (FDA) had accepted Vertex’s application for the new triple combination of vanzacaftor, tezacaftor and deivacaftor for the treatment of Cystic Fibrosis and was expected to issue its decision within 6 months, by 2 January 2025.
Approval of the new CFTR modifier ALYFTREK
As announced yesterday by Vertex, the US Food and Drug Administration (FDA) has approved the launch in the US of the new triple combination of vanzacaftor, tezacaftor and deivacaftor under the brand name Alyftrek. Alyftrek was approved for patients with Cystic Fibrosis aged 6 years and older who are eligible for Trikafta. Also for patients with 31 other rare mutations who could not previously receive any other innovative CFTR modulator therapy (CFTR Modulators).
Alyftrek offers an important new option for patients with Cystic Fibrosis, especially for those who cannot tolerate Trikafta. In addition, it is taken only once a day – as opposed to twice a day for other CFTR modifiers – making it easier for patients to take.
“ALYFTREK is the fifth CFTR regulator to receive FDA approval and represents another important milestone in our efforts to continuously innovate and improve the lives of people living with Cystic Fibrosis,” said Dr. Reshma Kewalramani, CEO and President of Vertex. “Our driver for more than 20 years has been to address the root cause of Cystic Fibrosis, cure more people with this disease and return more patients to normal levels of CFTR function. ALYFTREK, with its once-daily dosing, efficacy in 31 additional mutations and lower sweat chlorine levels than TRIKAFTA, is another step towards achieving this goal.”
31 rare mutations for Alyftrek
3199del6, G91R, M1101R, R560S, A559T, H199R, P99L, R560T, A559V, H609R, Q1100P, T604I, A561E, I1234Vdel6aa, Q452P, V520F, A613T, I1398S, R1066C, Y569C, A72D, I506T, R1066L, Y913C, D513G, L102R, R1066M
Expansion of Trikafta in the Americas
At the same time, the expansion of Trikafta in the Americas to the following 94 rare mutations was announced, expanding access to treatment to even more patients with rare mutations.
1507_1515del9, 2183A→G, A1067P, A107G, A309D, A62P, C491R, D1445N, D565G, D993Y, E116Q, E292K, F1107L, F200I, F587I, G1047R, G1123R, G1247R, G27E, G424S, G480S, G551A, G970S, H620P, H620Q, H939R;H949L, I105N, I125T, I148N, I331N, I506L, I556V, K162E, K464E, L1011S, L137P, L333F, L333H, L441P, L619S, M1137V, M150K, N1088D, N1303I, N186K, N187K, N418S, P140S, P499A, P750L, Q1313K, Q372H, Q493R, Q552P, R1048G, R117C;G576A;R668C, R297Q, R31C, R516S, R555G, R709Q, R75L, S1045Y, S108F, S1118F, S1235R, S549I, T1086I, T1246I, T1299I, T351I, V392G, V603F, Y301C, 2789+5G→A, 3272-26A→G, 3849+10kbC→T, N1303K, 711+3A→G, E831X, 5T;TG12, 5T;TG13, 296+28A→G, 621+3A→G, 1898+3A→G, 2789+2insA, 3850-3T→G, 3600G→A, 3849+4A→G, 3849+40A→G, 4005+2T→C, 1341G→A, 3041-15T→G, 2752-26A→G
See the relevant Press Releases of the pharmaceutical company Vertex here and here.
See the relevant announcements of the Cystic Fibrosis Foundation here and here.
Our Association is in constant communication and cooperation with the European Cystic Fibrosis Organisation CF Europe. We will inform you about anything new at European level that concerns Greece in the next web coffee break in January. The date and time will be announced after the holidays.
Stay tuned!
