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Kalydeco: Celebration In Australia

July 10, 2013

Vertex Pharmaceuticals announces on July 9, 2013, 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 250 people, or 8 percent of those with CF in Australia, have the G551D mutation.

Simon Bedson, Senior Vice President of International Commercial Operations for Vertex Pharmaceuticals commented on the Australian approval :

“The approval of Kalydeco in Australia is an important milestone for the CF community. The rapid review and approval by the TGA is a major step in making Kalydeco available for eligible Australians.”

Here are statistics on the size of the CF global population :

•   70,000 people worldwide
•     3,000 in Australia
•   30,000 in US
•   35,000 in Europe
•     4,000 in Canada.

Here are statistics about Australian CF patients:

•   53% have 2 copies of the F508del mutation, the most common mutation
•   40% have 1 copy    of the F508del mutation
•     8% 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. Reference a presentation made in June 2012 by Amy K. O’Sullivan, PhD, for a description of the detailed set of steps for the assessment and reimbursement of orphan drugs in Australia.

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.

Additional information regarding the reimbursement of Kalydeco in Australia is expected to be available later in 2013 following the publicly announced planned review of Kalydeco at the upcoming PBAC meeting, July 10 – 12, 2013. Currently, Kalydeco is not listed on the PBS.


“Orphan Drugs And Rare Diseases In Australia” Blog Post

Rare Voices Australia Organization.

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

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

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