Liquidia Corporation's Yutrepia (treprostinil) inhalation powder has been granted tentative approval by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). While the drug meets all regulatory standards for safety, quality, and efficacy, its market entry is delayed due to the regulatory exclusivity held by United Therapeutics for its competing product.
The FDA's decision means Yutrepia cannot be fully approved until May 23, 2025, when United Therapeutics' exclusivity for Tyvaso DPI expires. Liquidia's CEO, Roger Jeffs, expressed disappointment with the FDA's decision to grant exclusivity to United Therapeutics, stating the company plans to challenge the decision. Despite this setback, Jeffs remains optimistic about the "clear path to full approval of Yutrepia in both PAH and PH-ILD."
Clinical Trial Data
The tentative approval was supported by data from the Phase III INSPIRE trial. The study evaluated Yutrepia in both treprostinil-naïve patients and those transitioning from nebulized treprostinil. Results indicated that Yutrepia was safe and well-tolerated, irrespective of prior treprostinil exposure. Exploratory efficacy data suggested that patients receiving Yutrepia either stabilized or improved during the one-year treatment period.
Yutrepia's Mechanism and Formulation
Yutrepia is an inhaled dry-powder formulation of treprostinil, a vasodilator known for reducing high blood pressure in the pulmonary arteries and exerting anti-inflammatory effects. It is delivered via a palm-sized device, designed for ease of use. Liquidia's PRINT technology enables the creation of drug particles that are precise and uniform in size, shape, and composition, enhancing drug deposition in the lungs following inhalation.
Market Context and Competition
United Therapeutics' Remodulin, an injectable treprostinil, was the first FDA-approved treprostinil brand for PAH patients. Tyvaso, another treprostinil product from United Therapeutics, is delivered via oral inhalation and is indicated for both PAH and PAH-ILD. The FDA's decision to grant Tyvaso DPI a three-year regulatory exclusivity, which Liquidia is challenging, impacts the immediate market availability of Yutrepia.
Disease Burden and Unmet Needs
Pulmonary arterial hypertension (PAH) is a rare, chronic, progressive disease affecting an estimated 45,000 patients in the United States. Pulmonary hypertension associated with interstitial lung disease (PH-ILD) affects over 60,000 patients in the U.S. These conditions lead to right heart failure and death. Current treatments aim to alleviate symptoms, improve functional class, delay disease progression, and enhance quality of life, as there is currently no cure.
