The FDA on Monday only granted Liquidia’s Yutrepia tentative approval, keeping it off the U.S. market until after rival United Therapeutics’ exclusivity expires in May 2025.
The FDA on Monday signed off on Liquidia Corporation’s Yutrepia (nebulized treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. However, the regulator’s tentative approval means that Yutrepia cannot yet enter the U.S. market—despite meeting all regulatory standards of safety, quality and efficacy.
Liquidia must wait out the existing regulatory exclusivity of a competing product from United Therapeutics. The biopharma expects that the FDA will be able to grant traditional approval after May 23, 2025.
Despite the regulatory victory, investors were disappointed by the tentative approval. Liquidia’s shares dropped by almost 40% in pre-market trading on Monday.
Still, CEO Roger Jeffs struck a positive note in a statement, saying that the company is “pleased” with the FDA’s decision, which provides “a clear path to full approval of Yutrepia in both PAH [pulmonary arterial hypertension] and PH-ILD [pulmonary hypertension associated with interstitial lung disease].”
The tentative approval was backed by data from the Phase III INSPIRE trial, which found that Yutrepia was safe both in treprostinil-naïve patients and in those transitioning from other inhaled treprostinil treatments. INSPIRE also evaluated Yutrepia’s efficacy on an exploratory basis, showing that patients receiving Liquidia’s drug candidate either stabilized or improved through one year of treatment.
Yutrepia’s active ingredient is a nebulized formulation treprostinil, a vasodilator that is well-known for reducing abnormally high blood pressure while also exerting anti-inflammatory effects. The first FDA-approved treprostinil brand is United Therapeutics’ Remodulin, which is an injectable treatment for PAH patients of New York Heart Association Functional Class II to IV.
In July 2009, United Therapeutics won another FDA approval for Treprostinil, under the brand name Tyvaso. Like Remodulin, Tyvaso is indicated for PAH as well as PAH-ILD, with the goal of improving exercise capacity in treated patients. However, Tyvaso is delivered via oral inhalation using an accompanying device.
In giving Yutrepia only tentative approval, the FDA upheld Tyvaso’s three-year regulatory exclusivity, which was granted on May 23, 2022. Liquidia criticized this move, with Jeffs saying that “we are disappointed and disagree with the FDA’s decision to simultaneously grant regulatory exclusivity to United Therapeutics.”
This market exclusivity “encompasses chronic use of essentially any dry-powder formulation of treprostinil in the approved indications” according to Jeffs. Liquidia will “take quick action to challenge the FDA’s broad grant of regulatory exclusivity,” he said.
In June 2024, the U.S. District Court for the District of Delaware sided with Liquidia and blocked United Therapeutics’ motion for preliminary injunction, which sought to prevent the launch of Yutrepia. At the time, Liquidia said that the legal victory “reinforces the clear path” for the FDA to grant its application for Yutrepia.
