Patients with cystic fibrosis (CF) in the Republic of Ireland will be among the first in Europe to benefit from Vertex Pharmaceuticals’ triple combination regimen composed of elexacaftor (VX-445), tezacaftor, and Kalydeco (ivacaftor), upon its approval by the European Medicines Agency.
The triple combination regimen is currently being reviewed by the European Medicines Agency (EMA), and is awaiting approval for all indicated CF patients who are at least 12 years old. Of note, this triple regimen was recently approved in the United States, where it is sold as Trikafta.
The addition of this triple combination regimen to Ireland’s reimbursement agreement broadens the current agreement, and allows CF patients across Ireland who have specific mutations to access Vertex medications. The therapies included in the previous agreement are Kalydeco, Orkambi (lumacaftor/ivacaftor), and Symkevi (tezacaftor/ivacaftor, ivacaftor; sold as Symdeko in the U.S.).
Currently, CF patients of all ages with mutations included in Kalydeco’s marketing authorization — G551D, G551S, S549R, G1244E, S1251N, G1349D, S1255P, G178R or S549N — are eligible for treatment with Vertex medications.
Additionally, patients of any age who are homozygous (meaning both the maternal and paternal copies of the gene have the mutation) for the F508del mutation — the most common CF genetic defect — also have access to Vertex medications in Ireland.
Furthermore, patients of all ages who are heterozygous (only one of the gene copies has the mutation) for the F508del mutation and have another specific mutation (P67L, D579G, D1152H, R117C, 711+3A→G, 2789+5G→A, L206W, S945L, 3272-26A→G, R352Q, S977F, 3849+10kbC→T, A455E or R1070W) are able to receive Vertex medications.
Finally, heterozygous patients, 18 and older, who have the F508del mutation and the R117H mutation are also eligible for Vertex medications.
This newly amended long-term agreement will enable patients in Ireland to quickly access Vertex medications, in case the indications are extended to cover additional age groups and if new Vertex medicines are granted approval for these patient populations.
“With this expanded agreement, even more patients in the Republic of Ireland will be among the first in Europe to benefit from the triple combination treatment once the medicine is licensed,” Ludovic Fenaux, senior vice president of Vertex International, said in a press release.
Data supporting the company’s application to the EMA is based on results from two Phase 3 clinical trials, AURORA F/MF (NCT03525444), and AURORA F/F (NCT03525548), in CF patients 12 or older, which showed that patients given the triple combination therapy had significant improvements in lung function (primary endpoint of the studies), as well as in secondary endpoints, including a lower annual rate of pulmonary exacerbations, and reduced levels of chloride in patients’ sweat.
The triple combination treatment was also found to be generally well-tolerated.
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