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Vertex Pharmaceuticals: Kalydeco Delay In Australia

August 19, 2013

In July 2013, Vertex Pharmaceuticals announces that the Therapeutic Goods Administration (TGA) of Australia approves orphan drug Kalydeco (Ivacaftor). The approval is for patients with Cystic Fibrosis (CF) ages 6 and older, who have at least one copy of the G551D mutation in the CFTR gene. About 200 – 250 people, or approximately 8% of patients with CF in Australia, have the G551D mutation.

The Pharmaceutical Benefits Advisory Committee (PBAC) makes national funding decisions for the Australian public healthcare system.  The PBAC assesses orphan drugs separately from non-orphan drugs using very strict criteria. Australian approval and reimbursement of a new medicine is a multi-step process:

•   TGA approves the new medicine
•   Pharmaceutical Benefits Advisory Committee (PBAC) assesses the new medicine for effectiveness and cost-effectiveness
•   Listing on the Pharmaceutical Benefits Scheme (PBS) based on the PBAC assessment.

It was announced last week, that the PBAC has deferred making a decision on the Government subsidization of Kalydeco for CF patients with the G551D mutation. According to the Cystic Fibrosis Australia website :

“Cystic Fibrosis Australia (CFA) has been advised that the PBAC has deferred making a decision on the Government subsidization of Kalydeco for people living with G551D. A deferral means that the PBAC was unable to make a decision to “recommend” or “reject” the Kalydeco reimbursement application and has requested further information and discussion with the sponsor company, Vertex. A deferral decision also means that Kalydeco cannot, at this point in time, be reviewed by the Lifesaving Drugs Program (LSDP) ….”.  

CFA goes on to say that a deferral is not a rejection and that history shows that therapies deferred by the PBAC ultimately end up being approved at a later stage. Negotiations between the Government and Vertex Pharmaceuticals is expected to start shortly.   

The PBAC recommendation can be read on the Pharmaceutical Benefits Scheme website, starting the evening of August 23, 2013.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

  1. We have started a campaign to accelerate listing Kalydeco on the PBS, please follow to see how you can help. Thanks

    • Hi Gen –

      I will pass along the info via this Blog & Twitter about the campaign to accelerate the listing of Kalydeco on Australia’s PBS. Thanks for visiting the Blog & passing along the link. Thanks, Ann

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