FDA Approves Treprostinil Inhalation Powder for PAH, PH-ILD

The US Food and Drug Administration (FDA) approved treprostinil inhalation powder (YUTREPIA), a prostacyclin analog targeted to improve exercise capability for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD).1

Announced by Liquidia Corporation on May 23, 2025, regulatory approval was awarded based on positive data from the Phase 3 INSPIRE trial, in which treprostinil was shown to be safe and well-tolerated regardless of a patient’s previous exposure.

“I am so pleased that patients with PAH and PH-ILD now have this newly introduced option for inhaled treprostinil. Having treated patients for more than six years in Liquidia’s INSPIRE and extension studies, I am confident in the safety, tolerability and dosing that [treprostinil] offers,” said Nicholas Hill, chief of the pulmonary, critical care and sleep division and professor of medicine at Tufts University School of Medicine, and principal investigator on the Phase 3 INSPIRE study. “The low-effort inhalation device used to deliver [treprostinil] may make it easier to start and maintain patients on treatment, especially those with limited inspiratory flows or lung capacity.”

An estimated 45,000 patients with PAH have been diagnosed in the US. Current estimates suggest the PH-ILD prevalence in the United States is greater than 60,000 patients, although many ILD diseases are unknown due to underdiagnosis. Inhaled treprostinil solution (Tyvaso) was first approved by the FDA for PH-ILD in March 2021.2

With this approval, Treprostinil marked the first prostacyclin dry-powder formulation using Liquidia’s patented PRINT technology, designed to enhance deep-lung delivery with uniform, free-flowing particles in a low-effort device requiring lower inspiratory effort.

“PAH and PH-ILD impact more than 105,000 patients in the U.S. alone. These patient communities and the physicians who serve them need therapies that can lead to the improvement of quality of life,” said Matt Granato, president and chief executive officer of the Pulmonary Hypertension Association. “We are always glad to see industry research leading to the development of drugs that expand options for the patient community.”

The Phase 3, open-label, multicenter INSPIRE trial enrolled adults with PAH who transitioned to treprostinil inhalation powder from nebulized treprostinil or added treprostinil inhalation powder to prostacyclin-naive patients on ≤2 nonprostacyclin oral therapies. The primary safety outcomes were the incidence of adverse events (AEs) and serious AEs, followed by exploratory efficacy measures.3

With 121 patients enrolled in the trial, 29 discontinued due to AEs. Approximately 80% of the transition group and 96% of the prostacyclin naïve group were titrated to a dose ≥79.5 mcg four times daily at Day 360, respectively, with a single patient achieving a dose of 212 mcg. The most common AEs reported in the trial were cough, headache, upper respiratory tract infection, dyspnea, dizziness, throat irritation, diarrhea, chest discomfort, fatigue, and nasopharyngitis.

Although most events were considered treatment-related, investigators noted they were mild to moderate in severity and anticipated for prostacyclin therapy administered by inhalation. Exploratory efficacy analysis indicated patients remained stable or improved across the 1 year of treprostinil treatment.

Administration of treprostinil inhalation powder could offer clinicians and patients a preferred inhaled PAH therapy, with the opportunity for added convenience and a favorable safety profile. With the approval, Liquidia indicated its intent to launch treprostinil inhalation powder and inform physicians and patients of the potential unique benefits in the coming weeks.

“Today, we celebrate for the patients and physicians who will now have access to a potential best-in-class dry-powder form of treprostinil with exceptional portability, tolerability, titratability and durability,” said Roger Jeffs, chief executive officer of Liquidia. “Thank you to the clinical investigation team, our steering committee, and the members of the pulmonary hypertension patient communities who helped make this day a reality.”

References

1. U.S. FDA approves Liquidia’s YUTREPIATM (treprostinil) inhalation powder for patients with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Liquidia Corporation. May 23, 2025. Accessed May 23, 2025. https://www.liquidia.com/news-releases/news-release-details/us-fda-approves-liquidias-yutrepiatm-treprostinil-inhalation.

2. Kunzmann K. Treprostinil (Tyvaso) receives FDA approval for pulmonary hypertension linked to interstitial lung disease. HCP Live. April 17, 2023. Accessed May 23, 2025. https://www.hcplive.com/view/treprostinil-tyvaso-fda-approval-pulmonary-hypertension-linked-to-interstitial-lung-disease.

3. Hill NS, Feldman JP, Sahay S, et al. INSPIRE: Safety and tolerability of inhaled Yutrepia (treprostinil) in pulmonary arterial hypertension (PAH). Pulm Circ. 2022;12(3):e12119. Published 2022 Jul 1. doi:10.1002/pul2.12119