MORRISVILLE, N.C. — The U.S. Food and Drug Administration has granted Fast Track designation to Liquidia’s YUTREPIA for a potential new use treating Raynaud’s phenomenon associated with systemic sclerosis.
The designation covers YUTREPIA, or treprostinil inhalation powder, for patients with systemic-sclerosis-associated Raynaud’s phenomenon, often shortened to SSc-RP. The condition can cause recurring blood-vessel spasms, pain, numbness and color changes in fingers and toes.
Fast Track status is designed to increase communication between a drug developer and the FDA and can allow portions of an application to be reviewed as they become ready. It does not mean the agency has determined that the treatment is safe or effective for the proposed use, and it does not guarantee approval.
That distinction is important for YUTREPIA. The drug is approved to treat pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, but its use for SSc-RP remains investigational. Liquidia said safety and effectiveness for the new indication have not been evaluated by any regulatory authority.
The Morrisville biopharmaceutical company plans to study the treatment in RE-WARM, a Phase 2a randomized, open-label dose-finding trial. The ClinicalTrials.gov record identifies the study as NCT07748000.
Liquidia expects to enroll approximately 75 adults at as many as 30 U.S. sites. The company is targeting an October 2026 start and February 2027 primary completion, although those dates are projections and the trial had not begun when the designation was announced.
The study is designed primarily to characterize YUTREPIA’s safety and pharmacodynamic effects in the patient population. It also will explore whether treatment changes the number, severity and effect of Raynaud’s attacks. Results from an exploratory Phase 2a trial would not by themselves establish approval for the indication.
Liquidia estimates that about 30,000 U.S. patients have moderate-to-severe SSc-RP symptoms that could make them candidates for treatment. That addressable-population figure is a company estimate. Liquidia also says no therapy is currently approved by the FDA specifically for SSc-RP.
Chief Medical Officer Rajeev Saggar said the company views the designation as recognition of the condition’s seriousness and unmet treatment need. Liquidia did not announce jobs, capital investment or a commercialization timetable tied to the Fast Track decision.
The company disclosed the designation in a Sept. 3 announcement and a matching SEC-filed exhibit. The FDA explains the limits and potential benefits of the pathway on its Fast Track program page.
Sources:
ClinicalTrials.gov study record
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