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FDA Accepts Submission to Add PH-ILD to YUTREPIA Label
N.C., September 25, 2023 - 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 the FDA, YUTREPIA would
be indicated for the treatment of both PH-ILD and pulmonary arterial hypertension."
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
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 na ve 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
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 www.liquidia.com.
Tyvaso is a registered trademark of United Therapeutics
Cautionary Statements Regarding Forward-Looking
This press release may include forward-looking
statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release
other than statements of historical facts, including statements regarding our future results of operations and financial position, our
strategic and financial initiatives, our business strategy and plans and our objectives for future operations, are forward-looking statements.
Such forward-looking statements, including statements regarding clinical trials, clinical studies and other clinical work (including the
funding therefor, anticipated patient enrollment, safety data, study data, trial outcomes, timing or associated costs), regulatory applications
and related submission contents and timelines, including the potential for FDA approval of the NDA for YUTREPIA or any amendment to the
NDA for YUTREPIA, the timeline or outcome related to patent litigation in the U.S. District Court for the District of Delaware or inter
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