A groundbreaking global clinical trial has delivered promising results for a potential new treatment for idiopathic pulmonary fibrosis (IPF) and other forms of progressive pulmonary fibrosis (PPF), offering a major breakthrough for the pulmonary fibrosis (PF) community.
We welcome the full results of the FIBRONEER-IPFTM and FIBRONEER-ILDTM clinical trials that were published in the New England Journal of Medicine and presented this evening at the American Thoracic Society Congress in San Francisco. The studies tested a new oral medication called nerandomilast and results show it slowed the decline in lung function over one year in people living with IPF and other forms of pulmonary fibrosis — both chronic lung diseases with limited treatment options that deeply affect daily life.
This development could mark the first new treatment for over a decade, potentially transforming care for thousands of people across the UK and globally. The two current treatments, nintedanib and pirfenidone, were introduced more than 10 years ago and, while helpful in slowing disease progression, can cause significant side effects and are not suitable for everyone. Whilst the new drug doesn’t stop progression of disease, it offers hope as an additional treatment option that may be tolerated by more people.
Peter, living with PF and part of our Research Engagement Group said:
When you’re living with PF, you hold on to hope quietly. Progress takes time, and a lot of work happens behind the scenes — but this feels like a breakthrough we can actually see. A treatment that more people might tolerate could make a real difference to how we live and what comes next.
More than 2,200 people in over 40 countries took part across the trials for IPF and PPF. Participants who received nerandomilast experienced a significantly smaller decline in lung function compared to those who received a placebo. Importantly, the drug showed effectiveness both as a standalone therapy and when used alongside existing treatments.
While participants experienced some degree of lung function decline, reinforcing the need for ongoing research, the slowed progression seen in those taking nerandomilast is a substantial and encouraging step forward.
The most commonly reported side effect was diarrhoea. However, fewer than 1 in 25 participants stopped treatment because of it, indicating the drug was generally well tolerated. This is a significant difference compared to current treatments, where many have to stop taking the drug due to the impact of this and other side effects.
Daniel Saxton, CEO at APF said:
This milestone marks another step forward in the effort to overcome PF. Nerandomilast could be the first new approved treatment for PF in over 10 years, and it’s one that may be better tolerated and suitable for more people.
This provides fresh hope that it could soon become a widely available option for those living with this devastating disease. We are committed to advocating that regulators prioritise the review and approval of this medicine so people in the UK can access it as soon as possible.
What’s next?
People who completed the trial have been offered the opportunity to continue taking nerandomilast in a long-term follow-up study.
We are excited to see the impact of nerandomilast for IPF and other forms of PF. APF is committed to working closely with stakeholders, including the drug company and regulatory bodies, to ensure this potential treatment reaches the people who need them as soon as possible. The medication will still need to go through the drug approval processes in the UK before it is available.
We will continue to provide updates when we know more.
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