ORKAMBI - A NEW CFTR MODULATOR 

Treating the cause of CF 

Orkambi, the twice-a-day pill designed to treat people living with the most common mutation of cystic fibrosis (CF) - two copies of F508del - has been approved by the Food and Drug Administration (FDA) in the US.

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 in 2016 who are aged 12 and over. 

During clinical trials this break-through treatment has been shown to improve lung function by approximately 3%. Orkambi also reduced instances of chest infection, hospital stays and levels of lung damage.

Some side effects were identified and these included shortness of breath, upper respiratory tract infections, nausea, diarrhea and rash.

The 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, was born out of a long-term partnership between Vertex and the Cystic Fibrosis Foundation in the US. 

Vertex is planning on using a parallel approval process by engaging both the TGA and PBAC simultaneously. Orkambi is currently being assessed by the European Medicines Agency (EMA) and will be appraised by the NHS later this year.

Cystic Fibrosis Victoria and Cystic Fibrosis Australia plan to work with CF clinicians and Australian government regulatory agencies to improve access to treatments for patients with CF. We will be tracking the progress of Orkambi in Australia and overseas and providing regular updates in newsletters and on our website.