Cystic Fibrosis Australia can confirm the U.S. Food and Drug Administration (FDA) has approved Orkambi, the combination cystic fibrosis (CF) drug containing 400mg of lumacaftor and 250mg of ivacaftor (daily dose) for people who are 12 years of age and living with the F508del homozygous gene mutation.
Cystic Fibrosis Australia supports this decision and plans to continue the momentum of the Orkambi approval campaign here in Australia.
Two large phase-three clinical trials were conducted worldwide and 10 trial sites were located in Australia. FEV1 improvements were observed from Day 15, sustained throughout the 24-week trial and to 48 weeks in the open label follow up period. The mean improvement in lung function was approximately 3% though pulmonary exacerbations for patients on the drug combination were 39% less than the placebo group.
Importantly the drug combination not only initially improved FEV1. Orkambi trial participants also experienced weight gain, a reduction in hospitalisations and a reduction in the use of additional antibiotics.
The Vertex drug combination of ivacaftor (Kalydeco), which is designed to enhance the function of the CFTR protein once it reaches the cell surface and lumacaftor, which binds the defective CFTR and enables it to be transported to the surface of the cell, will improve the lives of many people living with CF.
Approximately 1,500 Australians have the F508del homozygous gene mutation and if approved by the TGA and PABC, Orkambi will be available to about 1000 of these people who are aged 12+ in 2016. Vertex is planning on using a parallel approval process by engaging both the TGA and PBAC simultaneously.
CFA is committed to maximising every opportunity to get the voice of the CF community heard and will be working with CF clinicians, and Australian government regulatory agencies to improve access to treatments for patients with CF. Cystic Fibrosis Australia will be tracking the progress of Orkambi in Australia and overseas and regular updates will be available at cysticfibrosis.org.au/cfa/orkambi.