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Submits Amendment to Add PH-ILD Indication to Tentatively Approved NDA for YUTREPIA (treprostinil) Inhalation Powder
N.C., July 26, 2022 - Liquidia Corporation (NASDAQ: LQDA) announced today the submission of an amendment to the
tentatively approved new drug application (NDA) for YUTREPIA (treprostinil) inhalation powder to add the treatment of pulmonary
hypertension associated with interstitial lung disease (PH-ILD). The U.S. Food and Drug Administration (FDA) has previously confirmed
in writing that the addition of the PH-ILD indication will not require any new clinical information. Upon acceptance of the amendment,
the FDA will confirm the type of resubmission as Class 1 or Class 2. If approved by FDA, YUTREPIA would be indicated for the
treatment of both PH-ILD and pulmonary arterial hypertension (PAH). The FDA can grant final approval of the PH-ILD indication in the
YUTREPIA label after the new clinical investigation exclusivity granted to Tyvaso expires on March 31, 2024.
with the amendment, Liquidia recertified under 21 U.S.C. 355(b)(2)(A)(iv), also referred to as paragraph IV certification, that the patents
listed for Tyvaso in the FDA's publication of Approved Drug Products with Therapeutic Equivalence Evaluations,
commonly known as the Orange Book, are invalid and/or not infringed by YUTREPIA. All Orange Book patents previously asserted by United
Therapeutics have already been found to be invalid or not-infringed as decided by U.S. District Court, confirmed on appeal, or by the
Patent Trial and Appeal Board (PTAB), pending appeal.
Jeffs, Chief Executive Officer of Liquidia, said: "We have submitted this request to ensure that patients may access YUTREPIA for
both the PAH and PH-ILD indications as soon as possible. We continue to hear from the community that alternative inhaled products are
needed, especially for those patients who may benefit from a low resistance dry powder inhaler that can be easily titrated to optimal
therapeutic effect like YUTREPIA. This feedback reinforces our undeterred commitment to deliver on the full potential of YUTREPIA to
improve patients' lives."
YUTREPIA (treprostinil) inhalation powder
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, which is indicated 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.
pulmonary arterial hypertension (PAH)
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.
pulmonary hypertension associated with interstitial lung disease (PH-ILD)
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 3-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.
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.
is a registered trademarks of United Therapeutics Corporation.
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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 final
FDA approval of the NDA for YUTREPIA, the timeline or outcome related to appeals arising from our patent litigation in the U.S. District
Court for the District of Delaware or inter partes review proceedings conducted at the PTAB, the issuance of patents by the USPTO
and our ability to execute on our strategic or financial initiatives, involve significant risks and uncertainties and actual results
could differ materially from those expressed or implied herein. The favorable decisions of the PTAB in the IPR for the '793 patent
and of the Court and CAFC in the Hatch-Waxman litigation are not determinative of the outcome of any appeal of those decisions. The words
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Chief Business Officer