OR WAIT null SECS
FDA Accepts Treprostinil Resubmission for PAH, PH-ILD Final Approval
Treprostinil (YUTREPIA) was previously granted tentative approval on August 16, 2024.
The US Food and Drug Administration (FDA) has accepted Liquidia’s new drug application (NDA) resubmission for Treprostinil (YUTREPIA) inhalation powder to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), with a Prescription Drug User Fee Act (PDUFA) goal date of May 24, 2025, for final approval.1
“We are pleased that the FDA has responded promptly to the resubmission that we filed on Monday. We look forward to working with the FDA over the coming months as we seek final approval for YUTREPIA and, in the meantime, will continue preparations to support a launch of YUTREPIA as soon as possible,” Roger Jeffs, PhD, Chief Executive Officer of Liquidia, said in a statement.1
The FDA previously granted tentative approval to Treprostinil for treating both PAH and PH-ILD on August 19, 2024, indicating that the therapy had met all regulatory standards for quality, safety and efficacy required but must await the expiration of regulatory exclusivity of a competing product before being granted final approval.2 The competing product in question, Tyvaso DPI, is a different formulation of Treprostinil whose 3-year regulatory exclusivity expires on May 23, 2025.
“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. 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. 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,” Jeffs said in a statement when the FDA granted tentative approval.2
Supporting data on Treprostinil are from the phase 3 INSPIRE trial which demonstrated efficacy and manageable safety in patients who were naïve to treprostinil, as well as those transitioning to YUTREPIA from nebulized treprostinil.
The trial enrolled 121 participants, of which 29 patients discontinued from the trial, with the most common reason being adverse events (AEs). Eighty percent of the Transition group and 96% of the prostacyclin naïve group titrated to a dose ≥79.5 mcg QID at Day 360, respectively, with one patient achieving a dose of 212-mcg QID.3
Most AEs were treatment-related, although mild-to-moderate in severity, and were expected for prostacyclin therapy administered by inhalation. The most common AEs were cough, headache, upper respiratory tract infection, dyspnea, dizziness, throat irritation, diarrhea, chest discomfort, fatigue, and nasopharyngitis.
