It’s an exciting time in the global mito community, as we’re seeing the first approvals for new medicines for some types of mitochondrial disease (mito).
So what does this mean for Australians impacted by mito? And how can you be part of the progress?Â
What has recently happened?
Two new medicines for mito have been approved in the United States to treat specific, rare types of mito.
- Elamipretide (Forzinity) for Barth Syndrome (approved October 2025)
- Nucleoside therapy (Kygevvi) for thymidine kinase 2 deficiency, or TK2d (approved November 2025)
These approvals are a global milestone. Behind them are years of relentless research, clinical studies and the strength of families who never stopped hoping.
What this means for Australians with mito?
These medicines aren’t available here just yet. Approval by the US Food and Drug Administration (FDA) doesn’t mean automatic access in Australia.Â
If you, or someone you care for lives with either Barth Syndrome or TK2d, your mito specialist can help you understand what these new treatments might mean for you. They can also guide on whether any clinical trials may be available to you.
In Australia, we have a two-stage process to make new medicines accessible.
- First is registration involving a review by the Therapeutic Goods Administration (TGA)
- Secondly, reimbursement where the government considers whether to fund new medicines through the Pharmaceutical Benefits Scheme (PBS)
What is Mito Foundation doing to help?
At Mito Foundation, we’re doing everything we can to bring these new treatments to Australia.
We are working closely with the 2 medicines companies to encourage them to apply for registration and reimbursement in Australia. But the most powerful part of our work is connecting them with the Australians who are directly affected.
If you, or someone you care for lives with either Barth Syndrome or TK2d, we would love to hear from you. Your voice can help shape our advocacy and make sure these medicines are accessible here.
- Please email advocacy@mito.org.au and/or
- join the Mito Registry to stay informed and support progress.
What is happening for other types of mito?
We are confident that treatments for other types of mito are on their way. Around the world, more than 60 potential treatments for mito are in development. Clinical trials are underway—including some right here in Australia—and more are on the horizon. In addition to this, research in the lab on new treatment ideas, including gene therapies, continues to make progress. Every one brings us a step closer to a future with more options, more hope.
Mito Foundation is working to increase the number of clinical trials available to Australians. That means partnering with mito specialists and medicine companies to bring trials here—and making sure the community is ready to participate.
You can read more about our work here. Having Australians participate in clinical trials increases the evidence available to make decisions about new medicines in Australia.
Other types of research play an important role alongside clinical trials. These include studies into new ways of measuring the impact of mito on people’s lives and understanding more about how mito affects people differently over time. Mito Foundation continues to fund and support these important projects.
If you’re living with mito, the most important thing you can do right now is join the Mito Registry. It’s a simple step—but one that could help bring clinical trials and treatments to more people, faster.Â
You can help bring new treatments closer
Every day, mito steals moments, memories and milestones. But with your support, we can change the future.
Your donation helps fund research, advocate for access, and give families hope that tomorrow might be brighter.
Donate today and stand with Australians living with mito. Together, we can get closer to a cure.
