FDA approves treprostinil dry powder inhaler for treatment of PAH

The Food and Drug Administration (FDA) has granted approval to Tyvaso DPI (treprostinil) inhalation powder for treating pulmonary arterial hypertension (PAH; WHO Group 1) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability.

United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, announced today that the U.S. Food and Drug Administration (FDA) has approved Tyvaso DPI™ (treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability. Tyvaso DPI represents a new formulation and inhalation device for inhaled treprostinil and is the only dry powder inhaler approved by the FDA for use in PAH and PH-ILD.

"Tyvaso DPI is one of the easiest ways for patients to administer a prostacyclin, delivering the proven efficacy of treprostinil through a small inhaler that fits in the palm of the patient's hand," said Michael Benkowitz, President and Chief Operating Officer of United Therapeutics. "We look forward to launching this exciting new product, and the opportunity to introduce treprostinil to more patients with PAH and PH-ILD."

"Prostacyclin-based therapies are effective in treating PAH, and recent data from the INCREASE study has shown that inhaled treprostinil is an effective treatment for PH-ILD," said Shelley Shapiro, M.D., Ph.D. at the David Geffen UCLA School of Medicine Pulmonary Hypertension Program.

"The convenience and portability of Tyvaso DPI may make it an important new option for patients with WHO Group 1 PAH and with WHO Group 3 PH-ILD, with the potential for improving the quality of life for this patient population."

PAH is life-threatening high blood pressure in the arteries of the lungs, affecting the ability of the heart and lungs to work properly. PAH affects an estimated 45,000 patients in the United States. Interstitial lung disease (ILD) is a group of conditions in which marked scarring occurs within the lungs. It is often complicated by pulmonary hypertension (PH; high blood pressure in the lungs), which furthers symptoms and decreases survival.

PH is estimated to affect at least 15% of patients with early-stage ILD (approximately 30,000 PH-ILD patients in the United States) and may affect up to 86% of patients with more severe ILD. Tyvaso® (treprostinil) Inhalation Solution and Tyvaso DPI are the only therapies approved by the FDA to treat PH-ILD.

FDA approval of the new drug application for Tyvaso DPI is supported by data from BREEZE, an open label study of 51 PAH patients on a stable regimen of Tyvaso Inhalation Solution who were transitioned to Tyvaso DPI. In subjects with PAH, transition from Tyvaso Inhalation Solution to Tyvaso DPI demonstrated safety and tolerance during the three-week treatment phase with significant improvements in six-minute walk distance, device preference and satisfaction, and patient reported outcomes.

Top line data from BREEZE were issued in January 2021, efficacy data were presented in September 2021, long-term open-label safety data were published in April 2022, and additional long-term safety and efficacy data were presented in May 2022. See the Important Safety Information below under "About Tyvaso DPI".