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FDA Grants Tentative Approval for Liquidia’s Yutrepia Inhalation Powder for Treatment of PAH

November 8, 2021

The U.S. Food and Drug Administration granted tentative approval for Liquidia’s Yutrepia inhalation powder for the treatment of pulmonary arterial hypertension to improve exercise ability in adult patients with New York Heart Association Functional Class II-III symptoms.

Photo: Damian deGoa, CEO of Liquidia

Tentative approval indicates that Yutrepia has met all regulatory standards for quality, safety and efficacy required for approval in the United States.

Due to a regulatory stay pursuant to the Drug Price Competition and Patent Term Restoration Act (Hatch-Waxman Act), Yutrepia cannot yet be marketed in the United States. In June 2020, United Therapeutics filed a lawsuit against Liquidia for alleged infringement of three patents related to its drug Tyvaso. As a result, the FDA cannot give final approval of Yutrepia until the expiration of the regulatory stay on October 27, 2022, or earlier resolution or settlement of the ongoing litigation.

“The tentative approval for YUTREPIA is another step toward providing an important option for patients with PAH in the U.S.,” said Tushar Shah, chief medical officer of Liquidia. “We believe Yutrepia can improve the limitations of current nebulized therapies by allowing the administration of an expanded dose range of inhaled treprostinil using a proven, convenient, palm-sized device.” 

Yutrepia is an experimental, inhaled dry powder formulation of treprostinil delivered through a proven, convenient, palm-sized device. It was designed using Liquidia’s PRINT technology, which enables the development of drug particles that are precise and uniform in size, shape, and composition, and that are engineered for optimal deposition in the lung following oral inhalation. Liquidia has completed INSPIRE, an open-label, multi-center phase 3 clinical study of Yutrepia in patients diagnosed with PAH who are naïve to inhaled treprostinil or who are transitioning from Tyvaso (nebulized treprostinil).

The addressable market for inhaled treprostinil is significant and expected to grow. In 2020, United Therapeutics reported that its nebulized formulation of treprostinil indicated for PAH achieved sales of more than $480 million. The attributes of Yutrepia including ease-of-use, convenience, direct lung delivery, and higher dosage range may not only make Yutrepia a preference to nebulized therapy, but also an alternative to oral treatments, and possibly a treatment option to delay the use of parenteral therapies in PAH. There may also be future expansion opportunities for inhaled treprostinil into additional indications.

“This is a significant milestone for Liquidia. Not only does the tentative approval establish the safety and efficacy of Yutrepia for PAH patients but, in the process, we have validated our proprietary PRINT technology to engineer discrete drug particles with uniform composition, size, and shape,” said Damian deGoa, CEO of Liquidia. “There is more work to be done. We will now focus our efforts on pre-commercial launch activities and the growing market opportunity for Yutrepia in PAH and potential new indications.”

Author: Rare Daily Staff

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