FDA Accepts Submission to Add PH-ILD to YUTREPIA™ Label

• Sets PDUFA goal date of January 24, 2024
• If approved, YUTREPIA would be indicated for treatment of both PAH and PH-ILD

MORRISVILLE, N.C., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Liquidia Corporation (the Company) (NASDAQ: LQDA) announced today that the U.S. Food and Drug Administration (FDA) accepted for review the Company’s amendment to the tentatively approved new drug application (NDA) for YUTREPIA™ (treprostinil) inhalation powder in which the Company is seeking to add the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD) to the label. The FDA confirmed the type of resubmission as Class II and has set a Prescription Drug User Fee Act (PDUFA) goal date of January 24, 2024.

Dr. Rajeev Saggar, Chief Medical Officer of Liquidia, said: “We are pleased that the FDA has accepted the submission for review with a PDUFA goal date well in advance of the expiration in March 2024 of the new clinical investigation exclusivity granted to Tyvaso®. If this amendment is approved by FDA, YUTREPIA would be indicated for the treatment of both PH-ILD and pulmonary arterial hypertension (PAH). “  

The FDA tentatively approved YUTREPIA to treat PAH in November 2021 and confirmed that the addition of the PH-ILD indication will not require any new clinical studies. The launch of YUTREPIA in both indications remains subject to the successful resolution of the ongoing litigation with United Therapeutics and final FDA approval. Additionally, the FDA may not grant final approval of the PH-ILD indication until after the new clinical investigation exclusivity granted to Tyvaso expires on March 31, 2024.

About YUTREPIA™ (treprostinil) inhalation powder

YUTREPIA is an investigational, inhaled dry powder formulation of treprostinil delivered through a convenient, low-resistance, palm-sized device. On November 5, 2021, the FDA issued a tentative approval for YUTREPIA for the treatment of pulmonary arterial hypertension (PAH) to improve exercise ability in adult patients with New York Heart Association (NYHA) Functional Class II-III symptoms. The FDA has confirmed that YUTREPIA may add the indication to treat pulmonary hypertension with interstitial lung disease (PH-ILD) without additional clinical studies. YUTREPIA 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 has completed INSPIRE, or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, 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 was previously referred to as LIQ861 in investigational studies.

About pulmonary arterial hypertension (PAH)

Pulmonary arterial hypertension (PAH) is a rare, chronic, progressive disease caused by hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death. Currently, an estimated 45,000 patients are diagnosed and treated in the United States. There is currently no cure for PAH, so the goals of existing treatments are to alleviate symptoms, maintain or improve functional class, delay disease progression, and improve quality of life.

About pulmonary hypertension associated with interstitial lung disease (PH-ILD)

Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) includes a diverse collection of up to 150 different pulmonary diseases, including interstitial pulmonary fibrosis, chronic hypersensitivity pneumonitis, connective tissue disease related ILD, and chronic pulmonary fibrosis with emphysema (CPFE) among others. Any level of PH in ILD patients is associated with poor three-year survival. A current estimate of PH-ILD prevalence in the United States is greater than 60,000 patients, though population growth in many of these underlying ILD diseases is not yet known due to factors including underdiagnosis and lack of approved treatments until March 2021, when inhaled treprostinil was first approved for this indication.

About Liquidia Corporation

Liquidia Corporation is a biopharmaceutical company focused on the development and commercialization of products in pulmonary hypertension and other applications of its PRINT® Technology. The company operates through its two wholly owned subsidiaries, Liquidia Technologies, Inc. and Liquidia PAH, LLC. Liquidia Technologies has developed YUTREPIA™ (treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Liquidia Technologies is also developing L606, an investigational liposomal formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer, for use in North America. Liquidia PAH provides the commercialization for pharmaceutical products to treat pulmonary disease, such as generic Treprostinil Injection. For more information, please visit .

Tyvaso® is a registered trademark of United Therapeutics Corporation.

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