Liquida fails to obtain full approval for drug against pulmonary hypertension as competitor retains market exclusivity

The FDA on Monday logged out for Liquida Corporation's Yutrepia (nebulized treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. However, the regulatory agency's provisional approval means that Yutrepia cannot yet enter the U.S. market – even though it meets all regulatory standards for safety, quality and efficacy.

Liquida must await the existing regulatory exclusivity of a competing product from United Therapeutics. The biopharmaceutical company expects the FDA to be able to grant a traditional approval after May 23, 2025.

Despite the regulatory success, investors were disappointed with the preliminary approval. Liquida's shares fell by almost 40 percent in premarket trading on Monday.

Nevertheless, CEO Roger Jeffs struck a positive note in a statement, saying the company was “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 provisional approval was supported by data from INSPIRE Phase III studywhich concluded that Yutrepia was safe in both treprostinil-naïve patients and in patients switching from other inhaled treprostinil treatments. INSPIRE also evaluated the efficacy of Yutrepia on an exploratory basis and showed that patients receiving Liquida's drug candidates either stabilized or improved after one year of treatment.

Yutrepia's active ingredient is a nebulized formulation of treprostinil, a vasodilator known to reduce abnormally high blood pressure while exerting anti-inflammatory effects. The first FDA-approved brand of treprostinil is United Therapeutics' Remodulinan injectable treatment for PAH patients in New York Heart Association functional class II to IV.

In July 2009, United Therapeutics received another FDA approval for Treprostinilunder the brand name Tyvaso. Like Remodulin, Tyvaso is indicated for both PAH and PAH-ILD, with the aim of improving the physical performance of the treated patients. Tyvaso is administered by oral inhalation using a companion device.

By granting only provisional approval to Yutrepia, the FDA confirmed Tyvaso's three-year regulatory exclusivity, which was granted on May 23, 2022. Liquida criticized this move. Jeffs said, “We are disappointed and disagree with the FDA's decision to grant regulatory exclusivity to United Therapeutics at the same time.”

This market exclusivity “covers the chronic use of virtually any dry powder formulation of treprostinil in the approved indications,” Jeffs said. Liquida will “take swift 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 ruled in favor of Liquida and repelled United Therapeutics' request for a preliminary injunction to block Yutrepia from coming to market. Liquida said at the time that the legal victory “reinforces the clear path” for the FDA to grant its request for Yutrepia.