A new agreement will give people in Scotland with cystic fibrosis access to the CF combination treatments Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) through the country’s health system, according to Vertex Pharmaceuticals, the company that sells both medicines.
The announcement follows a prior rejection by the Scottish Medicines Consortium (SMC) in August, in which the agency cited concerns that the cost of the treatments was not proportional to their health benefits.
Despite that first negative outcome, Vertex continued its efforts to ensure patient access through the health insurance system to the two approved therapies. The company has now reached a five-year agreement with SMC.
In addition to giving Scottish people with CF access to Orkambi and Symkevi at little or no cost, the agreement stipulates that Vertex will collect real-world data on the use of these medicines. That data should be used to support any future submissions to the SMC, the agency said.
“We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution,” Ludovic Fenaux, senior vice president of Vertex International, said in a press release. “It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi or Symkevi.”
Both Orkambi and Symkevi work by addressing the cause of CF, which is the lack of a working CFTR protein. The cystic fibrosis transmembrane conductance regulator (CFTR) protein helps to maintain the balance of salt and water on many surfaces in the body, such as the surface of the lungs. These medicines employ a one-two punch of getting the CFTR properly processed and placed where it is supposed to go, then keeping it stable and functioning properly once it is there.
Orkambi is specifically designed to reverse the effects of the most common CF mutation, the F508del mutation. Symkevi, known as Symdeko outside the E.U., is not specific to a particular CF-causing mutation. However, the tezacaftor/ivacaftor combination is designed to restore the CTFR protein’s function.
Both therapies contain ivacaftor — sold alone under the brand name Kalydeco — which helps stabilize the CFTR protein once it is at the surface of cells. The other components — lumacaftor in Orkambi and tezacaftor in Symkevi — are responsible for ensuring the correct folding of CFTR.
NHS Scotland estimates that about 900 people in Scotland have CF, and as many as 1 in 24 carry one mutation in the gene encoding CFTR.
Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.