The combination therapy for cystic fibrosis  – tezacaftor with ivacaftor (Symdeko) – is to be PBS reimbursed for patients 12 years and older who are homozygous for the F508del  mutation in the CFTR gene.

Symdeko will also be made available for people with CF who have one copy of the F508del mutation and another responsive residual function (RF) mutation in the CFTR gene.

In addition, the PBS listing of lumacaftor/ivacaftor (Orkambi) is to be expanded to include children aged 2-5 years with CF ages 2 to 5 who have two copies of the F508del mutation in the CFTR gene. Patients over the age of six have already been able to access Orkambi since October 2018.

While the PBS listings are scheduled to commence on 1 December,  patients will have access to Symdeko and Orkambi immediately due to the manufacturer Vertex agreeing to the Government’s request to provide early access.

The listings are expected to provide access to treatment for up to 1400 patients with CF, who might otherwise pay up to $250,000 a year for the drugs.

Symdeko was recommended for PBS subsidy by the PBAC at its March 2019 meeting but listing was delayed while negotiations on pricing were underway between the government and the manufacturer.

In its decision statement, the PBAC said the claim of superior efficacy compared with best supportive care (BSC) was supported in the short-term on the basis of the primary outcome in a study that indicated an increase from baseline in per cent predicted FEV1 of 6.8% after eight weeks of treatment, with this increase maintained for an additional 16 weeks of treatment.

However, the PBAC noted that it was unknown whether the improvements in ppFEV1 and in quality of life would be maintained over the long-term for the progressive lifelong disease. It was also unknown whether treatment would be associated with improvements in life expectancy, nutritional status (including weight and height) or reduction in pulmonary exacerbations or the need for other medications.