FDA approves Yutrepia, inhaled treprostinil, for PAH and PH-ILD
Liquidia’s dry powder formulation aims to improve exercise capacity
The U.S. Food and Drug Administration (FDA) has approved Yutrepia, Liquidia’s inhaled dry powder formulation of treprostinil, for improving exercise capacity in people with pulmonary hypertension (PH) associated with interstitial lung disease, known as PH-ILD, and pulmonary arterial hypertension, or PAH.
Yutrepia had previously held tentative approvals for these indications, but it could not yet be commercialized. This approval by the FDA allows the therapy to be marketed in the U.S.
Liquidia noted that the therapy is easy to titrate, meaning the dose can be slowly increased over time — key in personalizing medication. It also has shown durability, or continuing effectiveness over time, per the company.
“Today, we celebrate for the patients and physicians who will now have access to a potential best-in-class dry-powder form of treprostinil with exceptional portability, tolerability, titratability and durability,” Roger Jeffs, PhD, Liquidia’s CEO, said in a company press release.
Yutrepia’s list price will be $24,360 for a 28-day supply. The company is offering full support services to users, including training and demo kits, patient education materials, and financial assistance programs.
Liquidia noted that it remains committed to a commercial launch of Yutrepia in the coming weeks, despite ongoing legal issues with United Therapeutics. Those issues are related to patent protections surrounding United’s own inhaled dry powder formulation of treprostinil, called Tyvaso DPI.
“With today’s milestone, our commercial team is prepared to launch Yutrepia and bring meaningful change to the lives of patients in need,” Jeffs said, adding that the company “[looks] forward to speaking with physicians and patients about the unique benefits of Yutrepia in the days and weeks ahead.”
Yutrepia approval supported by Phase 3 INSPIRE trial
The FDA’s decision was welcomed by the U.S.-based Pulmonary Hypertension Association (PHA), with Matt Granato, the nonprofit’s CEO, noting that “PAH and PH-ILD impact more than 105,000 patients in the U.S. alone.”
Yutrepia’s approval marks progress toward a better treatment landscape for people with PAH and PH-ILD — both forms of pulmonary hypertension marked by elevated pressure in the blood vessels of the lungs.
“These patient communities and the physicians who serve them need therapies that can lead to the improvement of quality of life,” Granato said. “We are always glad to see industry research leading to development of drugs that expand options for the patient community.”
The active ingredient in Yutrepia is treprostinil, which works by mimicking the activity of a naturally occurring hormone called prostacyclin that prompts blood vessels to relax and widen. This makes blood flow easier and helps ease disease symptoms.
A few different formulations of treprostinil are already approved for treating forms of PH. Yutrepia is designed for deep lung delivery via a palm-sized inhalation device that’s used three to five times per day. Per the release, the device is “easy-to-use [and] low-effort … requiring less inspiratory effort.”
The approval was largely supported by data from a Phase 3 clinical trial called INSPIRE (NCT03399604) that showed that Yutrepia stabilized or improved exercise capacity and life quality in adults with PAH. The study involved patients who switched from Tyvaso, an inhaled treprostinil solution, or who were new to inhaled treprostinil.
Another ongoing study, dubbed ASCENT (NCT06129240), is further evaluating Yutrepia’s safety and efficacy in adults with PH-ILD.
Legal battles surround inhaled treprostinil formulations
Per its prescribing label, the most common side effects of Yutrepia are cough, headache, throat irritation, and dizziness.
Nicholas Hill, MD, of Tufts University School of Medicine, a principal investigator for the INSPIRE study, noted that “the low-effort inhalation device used to deliver Yutrepia may make it easier to start and maintain patients on treatment, especially those with limited inspiratory flows [inhalation rate] or lung capacity.”
Hill added: “I am so pleased that patients with PAH and PH-ILD now have this newly introduced option for inhaled treprostinil.”
The low-effort inhalation device used to deliver Yutrepia may make it easier to start and maintain patients on treatment. … I am so pleased that patients with PAH and PH-ILD now have this newly introduced option for inhaled treprostinil.
The therapy had previously earned tentative approvals for treating PH-ILD and PAH — meaning Yutrepia had satisfied the FDA’s quality, safety, and efficacy criteria.
However, it could not be brought to market because of patent protections for United’s inhaled treprostinil formulations. Legal battles surrounding the matter have been ongoing, but earlier this year, the FDA accepted an application from Liquidia seeking Yutrepia’s approval.
More recently, United filed a complaint against Liquidia alleging patent infringement, and is seeking to block the commercialization of Yutrepia for PAH and PH-ILD. The motion remains pending.
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