Vertex opens new doors for cystic fibrosis treatment in young children

Pharma News: In a monumental stride forward for the cystic fibrosis (CF) community, Vertex Pharmaceuticals has received a nod of approval from the Therapeutic Goods Administration (TGA) to expand the usage of Orkambi (lumacaftor/ivacaftor). This announcement positions Orkambi as the sole modulator therapy accessible to this exceptionally young age group.

The newly granted approval signifies a significant breakthrough for approximately 35 children in Australia, all of whom possess two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These children will now have the opportunity to receive a medicine that specifically targets the root cause of their CF, instead of merely managing symptoms.

Sabrina Barbic, the Senior Country Manager at Vertex ANZ, emphasised the critical importance of early intervention, stating, “Symptoms and organ damage start very early in the lives of people with cystic fibrosis. Treating patients as young as possible is important, as it can potentially slow the progression of this devastating disease.” Ms Barbic’s sentiments highlight the urgency and significance of providing effective treatment options for children at the earliest possible stage of their lives.

This TGA approval was made possible through Access Consortium, a virtual international work-sharing consortium comprising Australia, United Kingdom, Canada, Singapore and Switzerland. The successful collaboration involved a crucial partnership with Health Canada and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).

In other remarkable news, it has been one month since Cystic Fibrosis Australia, alongside members of the CF community and their supporters, attended a press conference where Health Minister Mark Butler announced that Trikafta was being added to the Pharmaceutical Benefits Scheme (PBS), making it accessible to eligible individuals within the age group of 6 to 11 years old.

This announcement sparked an outpouring of gratitude from Cystic Fibrosis Australia, who expressed their appreciation to all stakeholders involved in accelerating this listing, including Mark Butler MP and Vertex Pharmaceuticals. The organization also extended their gratitude to Dr Michael Freelander MP, Dr Gordon Reid MP, and Ryan Park MP for their unwavering commitment to the CF community and attending the momentous announcement.

“It is a testament to the power of advocacy and community effort that access to life-changing treatments is being extended to CF children,” said Cystic Fibrosis Australia CEO, Jo Armstrong.

With just one week remaining for community input for Orkambi, the patient advocacy group is urging the public to submit their consumer comments to the Pharmaceutical Benefits Advisory Committee (PBAC) ahead of the July PBAC agenda for consideration. This presents a unique opportunity for individuals to contribute to the decision-making process and shape the future of CF treatment accessibility in Australia.

With Vertex’s Orkambi and Trikafta at the forefront, children of increasingly younger ages are being granted the gift of hope and a chance at a brighter, healthier future.

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