Orkambi

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.

In March of this year Vertex received Australian Approval for ORKAMBI® (lumacaftor/ivacaftor) for people aged  12 and Older with Two Copies of the F508del Mutation.

You can read the full Business Wire press release here

Trials for 6+ years are currently underway.