FDA agrees to review approved inhaled therapy for use in pulmonary fibrosis
Developer says decision on Tyvaso for IPF expected by late April
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The U.S. Food and Drug Administration (FDA) will decide by early next year whether or not to expand its approval of nebulized Tyvaso (treprostinil) — an inhaled treatment for people with certain lung diseases — to allow its use in idiopathic pulmonary fibrosis (IPF), a respiratory condition marked by scarring, or fibrosis, in the lungs.
A decision is expected by late April, developer United Therapeutics said in a company press release announcing the FDA’s review of its supplemental new drug application (sNDA) for the therapy, administered via a hand-held device. According to United, an approval could make Tyvaso the first inhaled antifibrotic treatment available to people with IPF in the U.S.
United’s said its filing with the FDA was based on combined data from two completed late-stage clinical trials: TETON-1 (NCT04708782) and TETON-2 (NCT05255991). In both Phase 3 studies — which together enrolled more than 1,000 adults with IPF — Tyvaso met the goal of outperforming a placebo at preserving lung function and preventing clinical worsening.
“We’re galvanized by the FDA’s acceptance of the nebulized Tyvaso sNDA for review and believe the strength and rigor of our TETON clinical program support an approvable application for this differentiated multi-pathway treatment for patients managing this life-threatening disease,” said Martine Rothblatt, PhD, United’s chairperson and CEO.
The company noted that the FDA has awarded Tyvaso orphan drug status for IPF. This designation aims to incentivize the development of treatments for rare diseases, which will mean that Tyvaso would be granted seven years of market exclusivity in the U.S. if approved.
IPF — in which fibrosis occurs for unknown reasons — causes the lungs to become gradually fibrotic (scarred) and inflamed, making them less able to transfer oxygen into the blood. Among other symptoms, IPF makes it difficult to breathe. It is the most common form of idiopathic interstitial lung disease.
Tyvaso aims to open up lung blood vessels in IPF
Tyvaso works by mimicking prostacyclin, a naturally occurring molecule that helps widen blood vessels and prevents excessive fibrosis and inflammation. When inhaled directly into the lungs, the therapy is expected to help open up the blood vessels in the lungs, which could reduce blood pressure and improve blood flow.
“Nebulized Tyvaso combines direct lung delivery with multimodal activity across fibrotic, vascular [blood vessel], and inflammatory pathways that are central to IPF, with the potential to help address a significant unmet need for this life-threatening disease,” Rothblatt said.
In the U.S., Tyvaso is approved to boost exercise ability in people with both pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung diseases. Tyvaso was found to improve forced vital capacity (FVC) — the amount of air that can be forcefully exhaled after a deep breath — in patients with interstitial lung diseases, supporting further clinical testing in IPF, according to its developer.
Inhaled therapy showed benefits in 2 large clinical trials
TETON-1 involved nearly 600 participants ages 18 and older with IPF in the U.S. and Canada, while TETON-2 enrolled a similar number of adults with IPF in other countries. The main goal of both clinical trials was to measure the change in absolute FVC from the start of treatment to week 52, or essentially over one year. Other measures included the time until clinical worsening and the time until the first acute exacerbation, or sudden worsening of symptoms.
When data from the two clinical trials were combined, participants given nebulized Tyvaso had a significant improvement in absolute FVC compared with those receiving a placebo. After 52 weeks, the combined data also showed significant benefits for most other measures. These included a lower risk of clinical worsening and a lower risk of an acute exacerbation.
Participants also showed changes in percent-predicted FVC, the King’s Brief Interstitial Lung Disease score — a measure of quality of life — and diffusion capacity of the lungs for carbon monoxide (DLCO), a measure of how effectively the lungs transfer oxygen from the air into the bloodstream.
Those completing TETON-1 or TETON-2 could enter the ongoing open-label extension, called TETON-OLE (NCT04905693), in which all participants are receiving Tyvaso. Another clinical trial, called TETON-PPF (NCT05943535), is testing nebulized Tyvaso in progressive pulmonary fibrosis, a disease in which scarring continues to worsen in the lungs over time. Enrollment in TETON-PPF has been completed.
Anjula Bean
I cannot wait for this to be approved in U.S. and Canada . Our life depends on the new meds to improve this condition.
Charles Gary
Will it help shortness of breath?
Bob Matthews
Does Tyvsco reverse the effects of Idiopathic Pulmonary Fibrosis rather than just slow progression of the disease?
Rolando lima
When start with this medicine
Joan Flynn
Was this inhaler approved this April 2026 or are they saying next April 2027??
Doris farley
Are there any requirements for getting this medicine once it is approved. ? I ask this because my doctor told me that I could not get jasyco because of a particular requirement I couldn't meet.
Fridrik Tiedemann
I have IPF AND I am very interested in any medication or help in mitigating this terrible disease. Thank you !