Update on the PBAC review of ORKAMBI 200/125 (lumacaftor 200 mg/ivacaftor 125 mg) in Australia

Vertex is disappointed that ORKAMBI 200/125® (lumacaftor 200 mg/ivacaftor 125 mg) has not been recommended for listing on Australia’s Pharmaceutical Benefits Scheme (PBS) despite recognition by the Pharmaceutical Benefits Advisory Committee (PBAC) of ORKAMBI’s “potential clinical value” and “the strong support for subsidised access” expressed by people with CF, their carers, patient organisations and physicians.

Vertex is proud of our track record of getting our medicines to patients. We are committed to continuing to work with the Australian government to make ORKAMBI available to all eligible Australians, just as we did with KALYDECO® (ivacaftor). To that end, we will make another submission for ORKAMBI as quickly as the process allows. Within the existing government process, the next opportunity for ORKAMBI to be considered is the November 2016 PBAC meeting. Unfortunately, this means the earliest possible access for patients is not until 2017.

As such, the approximately 1,000 Australians who could benefit from ORKAMBI and who currently do not have a medicine to treat the underlying cause of their cystic fibrosis (CF) are being forced to wait for access. Today’s outcome demonstrates the difficulties of developing and assessing medicines for small patient populations.

CF is a rare, life-shortening genetic disease that causes progressive damage to a number of organs throughout the body. In Australia, approximately half of people with CF die before the age of 27. ORKAMBI is the first medicine to treat the underlying cause of CF in people ages 12 and older who have two copies of the F508del mutation. In November 2015, the medicine was submitted for assessment via the government’s parallel regulatory and reimbursement process, designed to achieve more rapid patient access to new medicines. The Therapeutic Goods Administration (TGA) approved its use in Australia in March 2016.

During the PBAC’s public consultation process, there were approximately 600 submissions from people with cystic fibrosis, their friends, family and medical experts. Nine Australian CF clinics in four states participated in the ORKAMBI Phase 3 clinical trials.

ORKAMBI is now available to all eligible patients in France, Germany, Austria and the United States.

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