The Australian government’s decision resulted from the completion of an agreement with Orkambi’s manufacturer, Vertex Pharmaceuticals, and was supported by the recent positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC).
As part of Australia’s National Medicines Policy, the reimbursement agreement for Orkambi will cover all CF patients age 6 or older who have two copies of the F508del mutation on the CFTR gene — the most common genetic mutation among CF patients.
“We are delighted that lumacaftor/ivacaftor [Orkambi] will be made available to eligible CF patients in Australia,” Stuart Arbuckle, executive vice president and chief commercial officer at Vertex, said in a press release.
“We would like to recognize the PBAC for seeing the value of this medicine and acknowledge the Department of Health and the Minister for Health for their active engagement and willingness to finalize the agreement rapidly,” Arbuckle said.
With this approval about 1,300 CF patients in Australia will join thousands of patients across the world who already can be treated with Orkambi. This therapy is currently reimbursed in several countries including the United States, Austria, Denmark, Germany, Ireland, Italy, the Netherlands, and Sweden.
The F508del mutation is known to result in little-to-no production of functional CFTR protein. Orkambi was designed specifically to bypass the defect induced by this particular genetic mutation, increasing the amount of mature CFTR protein as well as enhancing its activity.
The treatment is available in tablets, typically taken twice a day.
More recently, the FDA approved the use of this therapy in children ages 2-5, making Orkambi the first medicine indicated for the treatment of this age group. Vertex is awaiting a decision from the European Medicines Agency (EMA) for a similar indication extension, which is expected during the first half of 2019.