Orkambi approved in Australia, will others follow?
Australia joins the United States, Germany, Austria, Denmark, Ireland, Italy, Sweden, and the Netherlands in offering reimbursement for Orkambi (lumacaftor/ivacaftor), a medicine currently used to treat those with cystic fibrosis (CF). Having a yearly price-tag of over $250K AUD, many families were unable to pay for the medications out of pocket. With the subsidy, the monthly cost is reduced from about $20,000 to just under $40.
About cystic fibrosis
Cystic fibrosis is an incurable genetic disease where the mucus thickens and builds up in the respiratory and digestive tract. Patients eligible for treatment with Orkambi (lumacaftor/ivacaftor) have a F508del mutation in the gene that codes for the protein called cystic fibrosis transmembrane conductance regulator (CFTR). They are unable to regulate the amount of water secreted by their lungs and stomach. Water lubricates the passage of mucus and foreign particles out of our lungs, and also allows food to be digested. Although sufferers don't have obvious outward indicators of having the disease, symptoms like difficulty breathing, coughing, digestive problems, fatigue and a high chance of lung infection are part of their daily lives. When treated with medicines like Orkambi (lumacaftor/ivacaftor), many patients see a marked difference in their ability to digest food, gain energy, and breathe freely.
Orkambi (lumacaftor/ivacaftor) has been approved in several countries for use in adults. Given the recent positive outcomes shown in children, the US Food and Drug Administration (FDA) has also approved it for treatment of children as young as 2 years old, so they too will have a higher chance to lead longer lives. As stated by John McNamara, MD, lead study researcher and medical director of the CF program at Children's Minnesota, “This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before".
Earlier this year, the FDA approved another medicine for CF called Symdeko (tezacaftor/ivacaftor). This provides another option for patients older than 12 years with two copies of the F508del mutation or who have at least one mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor.
Is Orkambi (lumacaftor/ivacaftor) as effective as initially thought?
Canada and the United Kingdom are among several countries who have not approved the medicine saying that its efficacy is unclear. In a press release, Dr. Elena Schneider, a researcher at the University of Melbourne, says that her study shows the combination of lumacaftor and ivacaftor may reduce the medicine's own overall efficacy. However, research continues in order to fully understand the effects of the combination on the body.
Orkambi in the United Kingdom
A 2017 UK analysis reported around 10,500 CF patients, with almost 91% having the F508del mutation. Per patient, Orkambi (lumacaftor/ivacaftor) now costs about £100,000 annually and is not reimbursed by the NHS.
Orkambi in Canada
Based on the latest findings from the Canadian Cystic Fibrosis Registry, roughly 4200 people in Canada have cystic fibrosis, and 88% carry the F508del mutation. The medicine costs around $250,000 CAD per patient and is not reimbursed by the Canadian government.
The UK has about twice the population of Canada, with roughly three times more registered cystic fibrosis patients and a similar incidence of the mutation. These two countries are among several others that are still waiting for a better deal from the manufacturer. Not much is known about the current pricing deals, but one thing is clear; with such a high price tag the cost of the medication is too expensive for the average household to pay out of pocket. Thanks to the reimbursement programme, 1200 families in Australia will be able to afford this medicine.
Where is Orkambi (lumacaftor/ivacaftor) available?
If Orkambi (lumacaftor/ivacaftor) is not approved or available in your country, it's possible to import medicines in most countries on a Named Patient basis. If you or a loved one is looking for a CF medicine that is not yet available, read our homepage to understand how our team can help. Our team delivers medicines on a Named Patient basis around the world every day, with a service that’s highly rated by doctors and patients.
- Product information for AusPAR Orkambi 200/125 lumacaftor/ivacaftor, [PDF]. September 2016.
- Lopes, Jose Marques. Orkambi Soon to Be Added to Public Health Plan for CF Patients in Australia. Cystic Fibrosis News Today. August 21, 2018.
- The Canadian Cystic Fibrosis Registry, 2016 Annual Data Report [PDF]. Cystic Fibrosis Canada. 2016
- Cystic Fibrosis strength in numbers, 2016 Annual Data Report [PDF]. UK Cystic Fibrosis Registry. 2016.
- Orkambi Approval History. Drugs.com. Accessed November 2018.
- FDA Clears CF Drug Orkambi for Children as Young as Age 2, Medscape. 2018.
- Cystic fibrosis: Company urged to lower cost of life-changing drug. BBC. 21 April 2018.
- Interview on 5AA with Leon Byner. Australian Department of Health. Accessed November 2018.
- Bentham Science Publishers. Cytochrome P450 3A4 induction: Lumacaftor versus ivacaftor?. Eureka Alert. 12 April 2018.
- FDA Approves New CFTR Modulator Treatment for Cystic Fibrosis. Cystic Fibrosis Foundation. 12 February 2018.