Tyvaso boosts lung function in IPF trial, per top-line data
Phase 3 study of inhalation treatment hits main goal
The inhalation therapy Tyvaso (treprostinil) significantly improved a measure of lung function for people with idiopathic pulmonary fibrosis (IPF) in a Phase 3 clinical trial.
The top-line result means that the Phase 3 TETON 2 clinical trial (NCT05255991) has met its main goal. Tyvaso’s developer, United Therapeutics, plans to use findings from the study and another Phase 3 trial to apply for approval.
“TETON-2’s successful outcome affirms the anti-fibrotic power of Tyvaso. We have unlocked new hope for patients with IPF and their families,” Martine Rothblatt, PhD, United’s chairperson and CEO, said in a company press release.
Tyvaso is approved in the U.S. to improve exercise capacity in people with pulmonary arterial hypertension (PAH) or pulmonary hypertension associated with interstitial lung diseases (PH-ILD). Both PAH and PH-ILD are disorders characterized by pulmonary hypertension, or elevated pressure in the vessels that carry blood from the heart through the lungs.
Treprostinil, Tyvaso’s active ingredient, is a lab-made molecule designed to mimic the activity of prostacyclin, which is produced naturally in the body. Prostacyclin widens and relaxes blood vessels, easing blood flow and blood pressure.
FVC values rise for patients taking Tyvaso
In the TETON 2 study, 597 adults with IPF, ages 40 and older, were randomly assigned to receive either Tyvaso or a placebo, taken daily via inhalation with a nebulizer. The study’s main goal was to assess the effect of treatment on forced vital capacity (FVC) after about a year. FVC is a common measure of lung function that assesses how much air someone can blow out in a single, forceful breath.
United Therapeutics said average FVC values were significantly higher — better — in patients given Tyvaso than in those who took the placebo, changing by 95.6 mL on average over the study period. Tyvaso outperformed the placebo at improving FVC across various subgroups, including smokers, non-smokers, and patients taking the approved IPF treatments Ofev (nintedanib) and/or pirfenidone (marketed as Esbriet, with generics available).
The company said secondary measures, such as the time to a first clinical worsening event and life quality measures, also generally favored Tyvaso over the placebo.
“These overwhelmingly positive data send a clear signal of the potential benefits of Tyvaso for patients with IPF,” said Peter Smith, senior vice president of product development at United Therapeutics and the lead for the TETON clinical trial program. “We are deeply grateful to every patient who participated in this important study and the investigators whose dedication made this milestone possible. These results have the potential to reshape the treatment of IPF, extending new opportunities to a much broader patient population than ever before.”
Safety data from TETON 2 were consistent with the known profile of Tyvaso. According to its prescribing information, common side effects of the treatment in PAH and PH-ILD include cough, headache, nausea, dizziness, flushing, throat irritation and pain, diarrhea, and syncope (loss of consciousness upon rising from a seated or lying position).
Steven Nathan, MD, the TETON steering committee chair and medical director of Inova Fairfax Hospital’s advanced lung disease and lung transplant programs, said the Phase 3 results “represent a major step forward, giving us hope for improving outcomes in patients who desperately need better options.”
“These unequivocally positive results included endpoints that were not attained in prior phase 3 IPF clinical trials — including, importantly, a difference in quality of life,” Nathan said. “It is also notable that most patients were already on standard of care anti-fibrotic therapy, which makes these results even more impressive. We are especially excited for our patients, as these results offer a renewed sense of hope.”
The company said additional results from TETON 2 will be presented at a scientific conference later this month. Meanwhile, an identically designed Phase 3 trial called TETON (NCT04708782) is ongoing, with results expected in the first half of 2026. United Therapeutics hopes to use findings from both TETON trials as a basis for applications seeking approval of Tyvaso for IPF.
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