FDA Approves Treprostinil Inhalation Powder to Expand Treatment for PAH and PH-ILD
The US Food and Drug Administration (FDA) has approved treprostinil inhalation powder (YUTREPIA), a prostacyclin analog designed to enhance exercise capacity in adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), offering a new therapeutic option for these conditions.
YUTREPIA is the first and only prostacyclin dry-powder formulation enabled by Liquidia’s proprietary PRINT™ technology, which yields uniform, free-flowing particles designed to enhance deep-lung delivery via an easy-to-use, low-effort device requiring less inspiratory effort.
Dr. Roger Jeffs, Chief Executive Officer of Liquidia, said: “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. 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. With today’s milestone, our commercial team is prepared to launch YUTREPIA and bring meaningful change to the lives of patients in need, and we look forward to speaking with physicians and patients about the unique benefits of YUTREPIA in the days and weeks ahead.”
The approval of YUTREPIA is based on findings from the Phase 3 INSPIRE trial which evaluated patients who were naïve to treprostinil, as well as those transitioning to YUTREPIA from nebulized treprostinil. YUTREPIA was shown to be safe and well-tolerated regardless of a patient’s previous exposure to treprostinil. Results from the INSPIRE study were published in the Pulmonary Circulation Journal in 2022 and the Vascular Pharmacology Journal in 2021. Please see the “Selected Safety Information” in the section entitled “About YUTREPIA™ (treprostinil) Inhalation Powder.”
Dr. Nicholas Hill, Chief Pulmonary, Critical Care & Sleep Division, Professor of Medicine at Tufts University School of Medicine and Principal Investigator on the Phase 3 INSPIRE study, said: “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 YUTREPIA offers. The low-effort inhalation device used to deliver YUTREPIA may make it easier to start and maintain patients on treatment, especially those with limited inspiratory flows or lung capacity.”
Matt Granato, President and Chief Executive Officer of the Pulmonary Hypertension Association, said: “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. We are always glad to see industry research leading to development of drugs that expand options for the patient community.”
About Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension (PAH) is a rare, chronic, progressive disease caused by narrowing, thickening or stiffening of the pulmonary arteries that can lead to right heart failure and eventually death. Currently, an estimated 45,000 patients are diagnosed and treated in the United States. There is currently no cure for PAH, so the goals of existing treatments are to alleviate symptoms, maintain or improve functional class, delay disease progression, and improve quality of life.
About Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) includes a diverse collection of up to 200 different pulmonary diseases, including interstitial pulmonary fibrosis, chronic hypersensitivity pneumonitis, connective tissue disease-related ILD, and chronic pulmonary fibrosis with emphysema (CPFE) among others. Any level of PH in ILD patients is associated with poor 3-year survival. A current estimate of PH-ILD prevalence in the United States is greater than 60,000 patients, though population size in many of these underlying ILD diseases is not yet known due to factors including underdiagnosis and lack of approved treatments until March 2021, when inhaled treprostinil was first approved for this indication.
About YUTREPIA™ (treprostinil) Inhalation Powder
YUTREPIA is an inhaled dry-powder formulation of treprostinil delivered through a convenient, low-effort, palm-sized device. YUTREPIA was designed using Liquidia’s PRINT® technology, which enables the development of drug particles that are precise and uniform in size, shape and composition, and that are engineered for enhanced deposition in the lung following oral inhalation. Liquidia has completed the INSPIRE trial , or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, an open-label, multi-center phase 3 clinical study of YUTREPIA in patients diagnosed with PAH who are naïve to inhaled treprostinil or who are transitioning from Tyvaso® (nebulized treprostinil). YUTREPIA is currently being studied in the ASCENT trial (, or An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in PH, with the objective of informing YUTREPIA’s dosing and tolerability profile in patients with PH-ILD. YUTREPIA was previously referred to as LIQ861 in investigational studies.
