Final approval of the therapy may occur after expiration of the 3-year regulatory exclusivity for Tyvaso DPI on May 23, 2025.
The FDA granted tentative approval of treprostinil (Yutrepia) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), according to a news release from developer Liqudia Corporation.1
Anatomy of human lungs / Shahidah - stock.adobe.com
The product’s tentative approval indicates that treprostinil has met all regulatory standards for quality, safety, and efficacy required for approval by the FDA. However, final approval is contingent upon the expiration of regulatory exclusivity for a competing product. Final approval for Yutrepia may come after the exclusivity period for United Therapeutics’ Tyvaso DPI ends on May 23, 2025.
About INSPIRE
Trial Name: Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil (INSPIRE)
Clinicaltrials.gov Identifier: NCT03399604
Sponsor: Liquidia Technologies, Inc.
Summary: The primary objective of this study is to evaluate the long-term safety and tolerability of LIQ861, a dry powder formulation of treprostinil, in patients with Pulmonary Arterial Hypertension (PAH).
“We are pleased that the FDA agreed that our NDA amendment last July was proper, providing a clear path to full approval of Yutrepia in both PAH and PH-ILD,” said Roger Jeffs, MD, PhD, chief executive officer of Liquidia Corporation, in the release.1 “However, we are disappointed and disagree with the FDA’s decision to simultaneously grant regulatory exclusivity to United Therapeutics for Tyvaso DPI that encompasses chronic use of essentially any dry-powder formulation of treprostinil in the approved indications for a 3-year period for its new dosage form approved on May 23, 2022.”
The tentative approval of treprostinil is supported by findings from the phase 3, open-label, multicenter INSPIRE (NCT03399604) trial. The study enrolled participants 18 years and older with PAH who were naïve to treprostinil, as well as those transitioning to Yutrepia from nebulized treprostinil. The trial focused on assessing the safety and tolerability of Yutrepia by monitoring adverse events (AEs).
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Of the 121 enrolled patients, 29 discontinued the trial, with the most common reason being AEs. Common AEs included cough, headache, upper respiratory tract infection, dyspnea, dizziness, throat irritation, diarrhea, chest discomfort, fatigue, and nasopharyngitis.
By day 360, 80% of patients in both the transition and prostacyclin-naïve group had reached a target dose of 79.5 mcg or more 4 times daily, with 1 patient achieving a dose of 212 mcg 4 times daily. A previous phase 1 trial (LTI-102) demonstrated that a 79.5 mcg dose of Yutrepia resulted in similar systemic exposure to tresprostinil as seen with 9 breaths of treprostinil inhalation solution, suggesting that Yutrepia could offer similar efficacy in a more convenient manner.3
An evaluation of exploratory efficacy measures also found that patients maintained stable or improved conditions over 1 year of treatment. Further, in patients who were naïve to treprostinil, as well as those transitioning to Yutrepia from nebulized treprostinil, Yutrepia was shown to be safe and well-tolerated regardless of a patient’s previous exposure to treprostinil.
Whereas inhaled treatments offer benefits for managing PAH, current options have limitations. These include the need for multiple inhalations and the use of complex devices that require daily time and maintenance, which can be especially inconvenient for working individuals or those needing to be away from home.
Yutrepia addresses these challenges with a compact, easy-to-use, concealable dry-powder device that delivers the required dose in just 2 breaths. In a patient satisfaction survey, 98.2% of patients in the transition group of the INSPIRE trial expressed their preference for Yutrepia versus their prior nebulized tresprostinil inhalation system, underscoring patients’ desire for simpler, less cumbersome inhalation systems.2
“A more conveniently delivered inhaled therapy such as Yutrepia offers patients and clinicians the benefits of established efficacy of the drug class with a favorable tolerability profile and may lead to long-term treatment adherence,” wrote investigators.2 “In some patients, a better tolerated inhaled option could be preferable, even when compared with oral medications.”
Liquidia Corporation has announced it will seek final approval of Yutrepia as early as possible.
“We plan to take quick action to challenge the FDA’s broad grant of regulatory exclusivity and defend the ability for patients to have access to Yutrepia with the least delay possible,” said Jeffs.1
READ MORE: Cardiology Resource Center
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