PBS expansion puts life-changing cystic fibrosis treatment within reach for most patients

Starting July 1, 2025, Trikafta‘s (elexacaftor/tezacaftor/ivacaftor and ivacaftor) funding under the Pharmaceutical Benefits Scheme (PBS) will expand to include cystic fibrosis patients aged 2 years and older with a responsive CFTR gene mutation. This expansion encompasses an additional 271 mutations and includes patients without two ‘Class I’ mutations.

“This expansion to Trikafta’s PBS listing is great news for hundreds of Australians who live with rare mutations of CF, including children, and their families. As a result, these people will be able to lead longer and better-quality lives,” said Mark Butler, Federal Minister for Health, NDIS and Disability.

Jo Armstrong, CEO Cystic Fibrosis Australia, commented “This decision places Australia as a global leader for equitable access to CF therapies, a position made possible by the government’s pragmatic approach to real-world data, in vitro studies, and clinical evidence. For those who’ve waited, hoped, and campaigned, this is more than a policy decision, it’s a transformational outcome.”

With this PBS funding expansion, more than 90% of Australians living with cystic fibrosis now have access to a CFTR modulator.

“Vertex has been committed to the cystic fibrosis community for more than 20 years. Our goal has always been to ensure that all those who can benefit from our medicines get access as quickly as possible, no matter their age or genotype. This expanded PBS listing, enabling funded access to Trikafta for additional, often ultra-rare mutations, underscores our unwavering commitment to patients,” said Sabrina Barbic, Senior Country Manager of Vertex Pharmaceuticals ANZ.

Meanwhile, the Australian Cystic Fibrosis Data Registry (ACFDR) team at Monash University is conducting a study on pregnancy outcomes in women using Trikafta. The study aims to collect data on maternal health and early childhood outcomes, comparing outcomes between those who were and were not on Trikafta during pregnancy. Funding for the registry is provided by Cystic Fibrosis Australia and Vertex.

In other developments, Vertex announced results from the Phase 1/2 portion of the FORWARD-101 clinical trial of zimislecel (VX-880), a stem cell therapy for people with type 1 diabetes (T1D) suffering from impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). These findings were presented at the American Diabetes Association (ADA) congress.

Zimislecel demonstrated significant promise in maintaining normal blood glucose levels, leading to an 83% reduction in the need for exogenous insulin within 12 months. The trial also successfully met its primary goal of eliminating SHEs while maintaining HbA1c levels below 7%. However, challenges remain, notably the requirement for immunosuppressants, which may limit its uptake among certain patient groups, along with risks of adverse events such as neutropenia and acute kidney injury.

Currently advancing to Phase 3, the FORWARD trial aims to complete enrollment and dosing of approximately 50 participants by the end of 2025. Vertex is also planning a study to evaluate zimislecel in adults with T1D who have undergone kidney transplants and are receiving immunosuppressive therapy.

Vertex acquired VX-880 through its acquisition of Semma Therapeutics for $950 million in 2019. The company noted that while two patient deaths were previously reported, these were determined to be unrelated to the therapy.

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