Doctors, patients and industry welcome expansion of cystic fibrosis drug on the PBS

Pharma News: Clinicians, Vertex Pharmaceuticals and Cystic Fibrosis Australia have welcomed the expansion of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) on the PBS (Pharmaceutical Benefits Scheme).

Starting August 1, Trikafta will be available for the treatment of cystic fibrosis (CF) in children aged 2 to 5 years who carry at least one F508del mutation in the CFTR gene, the most prevalent genetic mutation causing CF worldwide. This expansion will benefit approximately 290 children, including 100 children who will gain access to a modulator for the first time.

“Given it is a progressive disease, it is important to treat children with CF as early in life as possible. With this expanded reimbursement of Trikafta, we can now treat more children as young as two years with a medicine that addresses the underlying cause of the disease. This is very welcome news for these children, their families and the CF community,” said Dr Andrew Tai, Paediatric Respiratory and Sleep Medicine Physician, Head of Department, Women’s and Children’s Hospital Adelaide.

At just 4 years old, Heath lives with the challenges of cystic fibrosis, a genetic condition that affects the lungs and digestive system. However, in those four years, Heath and his family have benefited from an incredible care team, learned how to manage his CF with treatments and therapies, and will now have access to a modulator once listed on the PBS.

Parents, Ashley and Lucas, have been awaiting the PBS listing of Trikafta before he commences school to ensure he was equipped for all that is ahead of now. 

Jo Armstrong, CEO of Cystic Fibrosis Australia, said “It is evident that working together gets results. Thank you to everyone who has been a part of this great outcome, including our dedicated Federation Members who tirelessly work with Cystic Fibrosis Australia around the country.

“We extend our heartfelt thanks to the Health Department, Vertex Pharmaceuticals, and everyone involved in making this expansion possible. Our collective efforts have driven real change, especially for our youngest CF Warriors, and we should all take pride in this achievement today.”

This latest reimbursement will result in more than 90% of the 3,700 people living with CF in Australia qualifying for reimbursed access to a medicine that treats the underlying cause of their disease.

“We are delighted that the Australian Government has recognised the value of Trikafta and the need for access for these children in Australia. It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype,” said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals.

Cystic Fibrosis Australia will be broadcasting live via Facebook, from Parliament House at approximately 9am, featuring remarks from Federal Health Minister Mark Butler.

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