Orkambi approved in Denmark and Austria

Orkambi approved in Denmark and Austria

Denmark and Austria have joined a growing number of countries worldwide to approve Orkambi for reimbursement. It appears that the deals reached with Denmark and Austria by Vertex have similarities to the pipeline deal which was first pioneered in Ireland. As previously reported, the Irish ‘pipeline deal approach’ also shaped access deals for Orkambi in Sweden and the Netherlands.

The two CF drugs produced by Boston based Vertex and presently approved for use in Europe are Kalydeco and Orkambi. A third drug, Symkevi, is still going through the appraisal process in the European Medicines Agency (EMA) but the signs are hopeful for final EMA approval in early 2019. Symkevi may even be available in Ireland in the first quarter of 2019 as part of the Irish pipeline deal.

Denmark and Austria join a total of 9 countries in total around the world that provide their patients access to CFTR modulator therapies and which also includes Australia, Germany, Ireland, Italy, the Netherlands, Sweden and the United States of America.

Note: Kalydeco is designed to impact on about 12% of the CF population in Ireland and first became available in 2013 (for those with the G551D altered CF gene and similar).  Orkambi has the potential to impact on over 50% of the CF population, - once all age restrictions are lifted (Orkambi is currently licensed for those aged 6 and over). Orkambi was first approved in Ireland in 2017 and subsequently extended to 6-11 year olds in 2018, with a 100% uptake in the latter group.

These 2 drug therapies are the first CF drugs to treat the underlying cause of CF for those with the relevant CF genotypes, so they can accurately described as ‘innovative’ and ‘ground-breaking’.