A four-year fight has finally ended and the cystic fibrosis drug known as ‘Orkambi’ will now be available to people who have been diagnosed with the life-limiting condition.
In a long struggle, which included campaigners, patients, and charities either taking to the streets or to social media to protest, the transformative treatment has been approved by NHS England. The medication will now be available as part of a two-year managed access agreement with Vertex, the manufacturer.
A 2017 UK Cystic Fibrosis Registry Report revealed that there are 3,968 people in England, 350 people in Scotland, 169 people in Wales and 156 people in Northern Ireland who could benefit from Orkambi. The two-year agreement with Vertex means thousands who have been waiting, up to four years in some cases, for access to the medicine will now be able to use it.
Orkambi is now the second precision medicine licensed for use by those with cystic fibrosis, with Kalydeco (also known as ivacaftor) being approved for clinical use in 2012.
Cystic fibrosis causes fatal lung damage and around half of those who are diagnosed with the condition live to the age of 32.
Whilst conventional treatments target the symptoms of cystic fibrosis, Orkambi is able to focus on genetic F508del mutation. This improves lung function and helps with breathing difficulties; a side effect that affects 50% of those who have been diagnosed in the UK with cystic fibrosis.
Orkambi is known as a combination medicine, made up of both ivacaftor and lumacaftor. Lumacaftor improves the number of proteins able to reach the surface of cells in the body. Ivacaftor helps the chloride channels in the cells operate as effectively as possible. The combination of both lumacaftor and ivacaftor helps maintain a healthy balance of salt and water in the organs, especially the lungs.
A study, which collected data over 96-weeks identified that Orkambi can slow decline in lung function by 42% – the number one cause of death among people with cystic fibrosis.
Feedback from patients who have been prescribed Orkambi has been positive. While some say it still feels like they have cystic fibrosis, many feel stronger, more energetic and hopeful for the future.
Now the deal has finally been accepted it means those with cystic fibrosis ‘will walk easier, feel the warmth of the sun rays, see the amazing colours…and find that beautiful something at the end,’ and they’ll do that together. Click here to read more about the incredible impact Orkambi is having on people’s lives.
Posted on: Nov 21, 2019