Announcement • Sep 03

Liquidia Corporation Receives FDA Fast Track Designation For YUTREPIA For Raynaud's Phenomenon Associated With Systemic Sclerosis

Liquidia Corporation announced 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. 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. Liquidia plans to initiate RE-WARM, a Phase 2a dose-finding study of YUTREPIA in SSc-RP, in the fourth quarter of 2026. 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. 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. 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. 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. 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%). 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.

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