Liquidia Corporation (NASDAQ: LQDA) has announced today that the U.S. Food and Drug Administration (FDA) has granted tentative approval of Yutrepia™ (treprostinil) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). This tentative approval comes after the FDA confirmed that the amendment to add PH-ILD to the Yutrepia New Drug Application (NDA) was proper and that the application otherwise meets the requirements for approval under the Federal Food, Drug, and Cosmetic Act.
The FDA’s tentative approval is a significant development for Liquidia Corporation, as it indicates that Yutrepia has met all regulatory standards for quality, safety, and efficacy required for approval in the United States. However, the final approval of Yutrepia for PAH and PH-ILD may occur after the expiration of the 3-year regulatory exclusivity for Tyvaso DPI on May 23, 2025.
Dr. Roger Jeffs, Ph.D., Chief Executive Officer of Liquidia, expressed disappointment with the FDA’s decision to simultaneously grant regulatory exclusivity to United Therapeutics for Tyvaso DPI, encompassing chronic use of essentially any dry-powder formulation of treprostinil in the approved indications for a three-year period for its new dosage form approved on May 23, 2022. Liquidia Corporation plans to take quick action to challenge the FDA’s broad grant of regulatory exclusivity and defend the ability for patients to have access to Yutrepia with the least delay possible.
The tentative approval of Yutrepia is based on findings from the Phase 3 INSPIRE trial, which evaluated patients who were naïve to treprostinil, as well as those transitioning to Yutrepia from nebulized treprostinil. Yutrepia was shown to be safe and well-tolerated regardless of a patient’s previous exposure to treprostinil. Results from the INSPIRE study were published in the Pulmonary Circulation journal in 2022 and the Vascular Pharmacology journal in 2021. The FDA’s approval also confirms that the supporting information related to the manufacturing, testing, and supply chain of Yutrepia meets regulatory standards for quality and safety in accordance with Good Manufacturing Practices (GMP).
Liquidia Corporation remains committed to addressing the unmet needs of PAH and PH-ILD patients and will seek final approval of Yutrepia as early as possible.
Yutrepia is an inhaled dry-powder formulation of treprostinil delivered through a convenient, low-effort, 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 enhanced deposition in the lung following oral inhalation. Liquidia 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). Yutrepia is currently being studied in the ASCENT trial, an open-label prospective multicenter study to evaluate safety and tolerability of dry powder inhaled treprostinil in pulmonary hypertension, with the objective of informing Yutrepia’s dosing and tolerability profile in patients with PH-ILD. Yutrepia was previously referred to as LIQ861 in investigational studies.
Liquidia Corporation is a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, with a current focus on the development and commercialization of products in pulmonary hypertension and other applications of its proprietary Print® technology. The company is also developing L606, an investigational sustained-release formulation of treprostinil administered twice-daily with a next-generation nebulizer, and currently markets generic treprostinil injection for the treatment of PAH.
The FDA’s tentative approval of Yutrepia marks a significant milestone for Liquidia Corporation and its commitment to addressing the unmet needs of patients with rare cardiopulmonary diseases. Following these announcements, the company's shares moved 2.0%, and are now trading at a price of $14.11. For the full picture, make sure to review Liquidia's 8-K report.
