FDA授予Liquidia YUTREPIA治疗系统性硬化症相关雷诺现象的Fast Track资格认定

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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities
Exchange Act of 1934
Date of Report (Date of earliest event reported):
September 3, 2026
LIQUIDIA
CORPORATION
(Exact name
of registrant as specified in its charter)
Delaware
001-39724
85-1710962
(State or other jurisdiction
of incorporation)
(Commission
File Number)
(IRS Employer
Identification No.)
419
Davis Drive
,
Suite
100
,
Morrisville
,
North Carolina
27560
(Address of principal executive
offices)
(Zip Code)
Registrant’s telephone number, including
area code:
(
919
)
328-4400
(Former
name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K
filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (
see
General Instruction A.2. below):
¨
Written communications pursuant to Rule 425 under the Securities Act (17 CFR
230.425)
¨
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
¨
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange
Act (17 CFR 240.14d-2(b))
¨
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange
Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title
of each class
Trading
Symbol(s)
Name
of each exchange on which registered
Common
stock
LQDA
The
Nasdaq
Stock Market LLC
Indicate by check mark whether the registrant is
an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the
Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging
growth company
¨
If
an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying
with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
¨
Item 8.01
Other Events.
On September 3, 2026, Liquidia Corporation, a
Delaware corporation (the “Company”), issued a press release announcing that the U.S. Food and Drug Administration has granted
Fast Track Designation to YUTREPIA
®
(treprostinil) inhalation powder for the treatment of Raynaud’s phenomenon
associated with systemic sclerosis. A copy of the press release is filed as Exhibit 99.1 hereto and is incorporated by reference into
this Item 8.01.
Item 9.01
Financial Statements and Exhibits.
(d)
Exhibit
No.
Exhibit
99.1
Press Release of Liquidia Corporation, dated September 3, 2026.
104
Cover Page Interactive Data File (embedded within the Inline XBRL document).
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
September 3, 2026
Liquidia Corporation
By:
/s/ Michael Kaseta
Name: Michael Kaseta
Title: Chief Financial Officer and Chief Operating Officer

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EX-99.1
2
tm2624724d1_ex99-1.htm
EXHIBIT 99.1
Exhibit 99.1
FDA Grants Fast Track Designation to YUTREPIA® for the Treatment
of Raynaud's Phenomenon Associated with Systemic Sclerosis
·
Fast Track designation underscores the significant unmet need for patients
with systemic sclerosis-associated Raynaud's phenomenon (SSc-RP), a debilitating manifestation affecting up to 90% of SSc patients
·
No therapy is currently approved in the U.S. to treat SSc-RP and management
options are limited and often inadequate for patients with more severe disease
·
Liquidia plans to initiate RE-WARM, a Phase 2a dose-finding study of YUTREPIA
in SSc-RP, in the fourth quarter of 2026
MORRISVILLE, N.C., Sept. 3, 2026 (GLOBE NEWSWIRE) --
Liquidia
Corporation (Nasdaq: LQDA), a biopharmaceutical company driven by science and compassion to revolutionize care for patients with challenging
respiratory and vascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation
to YUTREPIA® (treprostinil) inhalation powder for the treatment of Raynaud's phenomenon associated with systemic sclerosis (SSc-RP).
The potential use of YUTREPIA in SSc-RP is currently under clinical development, and its safety and efficacy for this indication have
not been evaluated by any regulatory authority.
Systemic sclerosis (SSc) is a rare, chronic autoimmune disease marked
by widespread vasculopathy and fibrosis of the skin and internal organs, and it carries the highest mortality of any systemic rheumatic
disease. Raynaud's phenomenon (RP) consists of recurrent, often severe vasospastic episodes that cause pain, numbness and color change
in the fingers and toes. Affecting up to 90% of SSc patients, these attacks are more frequent, prolonged and severe than primary Raynaud's,
often progressing to digital ischemic ulcers in an estimated 40% to 60% of patients and, in recurrent cases, digital amputation. At a
2020 FDA Patient-Focused Drug Development meeting, SSc patients identified Raynaud's attacks as among the disease's most bothersome and
impactful symptoms, citing pain, functional limitation and considerable emotional distress tied to fear of ulceration and tissue loss.
A central driver of SSc-RP is deficient endogenous prostacyclin production, which contributes to the vasospasm, platelet activation and
vascular remodeling underlying the disease. Liquidia estimates the addressable population of SSc-RP patients with moderate to severe symptoms
to be approximately 30,000 patients in the United States.
Dr. Rajeev Saggar, Chief Medical Officer, said: "Fast Track designation
reflects the seriousness of this condition and the need for new options. No therapy is currently approved by the FDA specifically for
SSc-RP. We are encouraged by the opportunity to evaluate whether YUTREPIA can reduce the complications of moderate to severe Raynaud’s
that most affect patients' quality of life, and we are committed to advancing this program with urgency, starting with the RE-WARM study
later this year."
The FDA's Fast Track program is designed to facilitate the development
and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need. Fast Track designation provides
Liquidia with the opportunity for more frequent interactions with the FDA throughout development, the potential for rolling review of
a New Drug Application, and, if relevant criteria are met, potential eligibility for Priority Review and/or Accelerated Approval.
About RE-WARM
Liquidia plans to initiate RE-WARM (NCT07748000), a Phase 2a, randomized,
open-label, dose-finding study of YUTREPIA in approximately 75 adults with SSc experiencing symptomatic Raynaud's phenomenon attacks,
at up to approximately 30 sites in the United States. RE-WARM is designed to characterize the safety and pharmacodynamics of YUTREPIA
in this population. The study also explores whether treatment reduces the number, severity and impact of RP attacks. The study is expected
to begin in October 2026, with primary completion targeted for February 2027.
About YUTREPIA® (treprostinil) Inhalation Powder
YUTREPIA is an inhaled dry-powder formulation of treprostinil delivered
through a convenient, low-effort, palm-sized device. 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. YUTREPIA is approved for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension
associated with interstitial lung disease (PH-ILD). YUTREPIA is not approved for the treatment of Raynaud's phenomenon associated with
systemic sclerosis, and there is no guarantee that the FDA will ultimately approve YUTREPIA for this use, even with Fast Track designation.
INDICATION
YUTREPIA (treprostinil) inhalation powder is a prostacyclin analog
indicated for the treatment of:
·
Pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise ability.
Studies establishing effectiveness predominately included patients with NYHA Functional Class III symptoms and etiologies of idiopathic
or heritable PAH (56%) or PAH associated with connective tissue diseases (33%).
·
Pulmonary hypertension associated with interstitial lung disease (PH-ILD;
WHO Group 3) to improve exercise ability. The study establishing effectiveness predominately included patients with etiologies of idiopathic
interstitial pneumonia (IIP) (45%) inclusive of idiopathic pulmonary fibrosis (IPF), combined pulmonary fibrosis and emphysema (CPFE)
(25%), and WHO Group 3 connective tissue disease (22%).
SELECTED SAFETY INFORMATION: WARNINGS AND PRECAUTIONS
·
Treprostinil is a pulmonary and systemic vasodilator. In patients with low
systemic arterial pressure, treatment with treprostinil may produce symptomatic hypotension.
·
Treprostinil inhibits platelet aggregation and increases the risk of bleeding.
·
Co-administration of a cytochrome P450 (CYP) 2C8 enzyme inhibitor (e.g.,
gemfibrozil) may increase exposure (both Cmax and AUC) to treprostinil. Co-administration of a CYP2C8 enzyme inducer (e.g., rifampin)
may decrease exposure to treprostinil. Increased exposure is likely to increase adverse events associated with treprostinil administration,
whereas decreased exposure is likely to reduce clinical effectiveness.
·
Like other inhaled prostaglandins, YUTREPIA may cause acute bronchospasm.
Patients with asthma or chronic obstructive pulmonary disease (COPD), or other bronchial hyperreactivity, are at increased risk for bronchospasm.
Ensure that such patients are treated optimally for reactive airway disease prior to and during treatment.
·
Most common adverse reactions with YUTREPIA (≥10%) are cough, headache,
throat irritation and dizziness.
Prescribing Information and Instructions for Use for YUTREPIA (treprostinil)
inhalation powder are available at
https://www.yutrepia.com/full-prescribing-information.pdf
.
About Liquidia Corporation
Liquidia Corporation is a biopharmaceutical company driven by science
and compassion to revolutionize care for patients with challenging respiratory and vascular diseases. The company's current focus spans
the development and commercialization of products in pulmonary hypertension and other applications of its proprietary PRINT® Technology.
PRINT enabled the creation of YUTREPIA® (treprostinil) inhalation powder, a drug approved for the treatment of pulmonary arterial
hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). YUTREPIA is currently under development
for other indications, including SSc-RP. The company is also developing L606, an investigational extended-release formulation of treprostinil
administered twice daily with a next-generation nebulizer, and currently markets generic Treprostinil Injection for the treatment of PAH.
To learn more about Liquidia, please visit www.liquidia.com.
Cautionary Statements Regarding Forward-Looking Statements
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, the receipt, timing and continuation of Fast Track designation and any benefits thereof, and our ability
to successfully develop and, if approved, commercialize YUTREPIA for SSc-RP or any other product candidate, involve significant risks
and uncertainties and actual results could differ materially from those expressed or implied herein. Fast Track designation does not guarantee
that YUTREPIA will receive FDA approval for the treatment of SSc-RP, that development will proceed on the anticipated timeline, or that
RE-WARM or any future study will produce favorable results. The words “anticipate,” “believe,” “continue,”
“could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,”
“predict,” “project,” “should,” “target,” “would,” and similar expressions
are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations
and projections about future events and financial trends that we believe may affect our financial condition, results of operations, business
strategy, short-term and long-term business operations and objectives and financial needs. These forward-looking statements are subject
to a number of risks discussed in our filings with the SEC, as well as a number of uncertainties and assumptions. Moreover, we operate
in a very competitive and rapidly changing environment and our industry has inherent risks. New risks emerge from time to time. It is
not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which
any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements
we may make. In light of these risks, uncertainties and assumptions, the future events discussed in this press release may not occur and
actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Nothing in this
press release should be regarded as a representation by any person that these goals will be achieved, and we undertake no duty to update
our goals or to update or alter any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact Information
Investors:
Jason Adair
Chief Business Officer
919.328.4350
Media:
media@liquidia.com