The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) Wales have reached an agreement with Vertex Pharmaceuticals that will allow people with cystic fibrosis (CF) who live there to access all of Vertex’s currently approved medications for the disease.
Specifically, Vertex’s Orkambi (lumacaftor/ivacaftor) will be available for people with CF who are two years and older, and who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Symkevi (tezacaftor/ivacaftor combo, known as Symdeko in the United States) will be available for people with CF who are 12 and older, and who have two copies of the F508del mutation in CFTR, or who have one F508del mutation and one of the other 14 mutations for which Symkevi is licensed. (These are: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.)
Additionally, the new agreements expand access to Kalydeco (ivacaftor). The therapy now is available to people with CF who are 18 or older and have a R117H mutation in the CFTR gene, as well as to people with CF who are one year or older and have one of the following nine mutations in CFTR: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
The new agreements also extend to any therapeutic indications for which these medications are approved in the future.
All these Vertex therapies work by either increasing the ability of the CFTR protein to get into the cell membrane, which is where it needs to be to carry out its function, or by improving the functionality of the protein once it is in the proper location.
“We are pleased that the nearly 280 eligible cystic fibrosis patients in Northern Ireland will soon have access to CFTR modulators to treat the underlying cause of their disease, and we thank the authorities in Northern Ireland for their collaboration and commitment in this agreement,” Ludovic Fenaux, senior vice president at Vertex International, said in a press release.
Apart from Northern Ireland, this also is “good news for the approximately 270 eligible cystic fibrosis patients in Wales who will soon have access to CFTR modulators. … We thank the authorities in Wales for their collaboration in accepting this offer under the same terms as were recently announced in England,” Fenaux added in another press release.
Vertex hopes to have its CF therapies available for patients in Northern Ireland and Wales within the next few weeks.