The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for Tyvaso DPI (inhaled treprostinil) for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease.
Tyvaso DPI is an investigational drug-device combination therapy that consists of a dry powder formulation of treprostinil and a small, portable, dry powder inhaler. The Company believes that Tyvaso DPI will provide a more convenient method of administration compared with the traditional nebulized Tyvaso therapy.
The NDA submission is supported by data from the open-label phase 1 BREEZE study (ClinicalTrials.gov Identifier: NCT03950739) that evaluated the safety and tolerability of Tyvaso DPI in 51 adult patients with PAH on a stable regimen of Tyvaso inhalation solution. Findings demonstrated that the transition from Tyvaso inhalation solution to Tyvaso DPI was safe and well tolerated. There were no treatment-related serious adverse events. Most adverse events were mild to moderate in severity.
Additionally, a separate pharmacokinetic study evaluated 3 dose levels of Tyvaso DPI and Tyvaso inhalation solution in 36 healthy volunteers. Results showed that participants had comparable systemic treprostinil exposure for each corresponding Tyvaso DPI and Tyvaso inhalation solution dose level. The safety profile of Tyvaso DPI was consistent with that observed with Tyvaso inhalation solution.
A Prescription Drug User Fee Act (PDUFA) target date of October 2021 is expected for this application.
“The acceptance of the Tyvaso DPI NDA for review represents an important regulatory step toward offering this meaningful new product to both PAH and PH-ILD patients,” said Martine Rothblatt, PhD, Chairperson and CEO of United Therapeutics. “If approved, Tyvaso DPI will represent yet another path to help us achieve our goal of serving 25,000 patients by the end of 2025.”
- United Therapeutics announces FDA acceptance of Tyvaso DPI New Drug Application for Priority Review. [press release]. Silver Spring, MD and Research Triangle Park, NC: United Therapeutics Corporation; June 16, 2021.
- United Therapeutics announces BREEZE study of investigational Tyvaso DPI meets primary objective. [press release]. Silver Spring, MD and Research Triangle Park, NC: United Therapeutics Corporation; January 28, 2021.