Vertex, NHS England reach wide-ranging agreement for cystic fibrosis drugs Orkambi, Symkevi, Kalydeco

Vertex Pharmaceuticals on Thursday announced that it has concluded an access agreement with NHS England for all of the company's currently licensed cystic fibrosis medicines and any future indications of these drugs. According to Vertex, the deal means eligible patients in England will have access to Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor), as well as expanded access to Kalydeco (ivacaftor). 

"This important agreement, reached in collaboration and partnership with NHS England and the National Institute for Health and Care Excellence, will allow more than 5000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease," remarked Ludovic Fenaux, senior vice president of international commercial operations at Vertex. 

Specifically, cystic fibrosis patients in England ages two years and older who have two copies of the F508del mutation in the CFTR gene can receive Orkambi, while Symkevi in combination with Kalydeco can be prescribed to patients ages 12 years and older who are either homozygous for F508del or have one copy of the F508del mutation and one of the other 14 licensed mutations. The deal also offers expanded access to Kalydeco to include adults who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.

NHS England noted that the agreement comes with a binding condition that Vertex submit its full portfolio to NICE for comprehensive appraisal, including "in due course" the company's new triple therapy. That treatment, which combines Vertex's next-generation CFTR corrector elexacaftor with Symkevi, was approved this week in the US under the name Trikafta. "The company has also agreed to a flexible commercial mechanism which will ensure continued patient access to these three medicines following completion of the NICE appraisal," NHS England added. 

The news follows a decision by Vertex earlier this year to rejoin talks with NICE and NHS England in a bid to break a standoff regarding funding for Orkambi. NICE had come out against NHS coverage for Orkambi in 2016, citing lack of cost-effectiveness, with Vertex pulling out of negotiations last year after rejecting an NHS offer of £500 million pounds ($643 million) over five years to access Orkambi and its other cystic fibrosis drugs. 

NHS chief executive Simon Stevens said the new deal would apply to Wales and Northern Ireland as well, but not Scotland, which recently made its own access agreement with Vertex for Orkambi and Symkevi. "Six weeks ago a decision was taken there to step away from coordinated UK-wide approach by ignoring the independent expert medical advice of the Scottish Medicines Consortium," commented Stevens, but "we are legally able to share with the Scottish government confidential details of the improved deal we have now negotiated for England."

To read more Top Story articles, click here.