U.S. FDA Grants Tentative Approval of YUTREPIA™ (treprostinil) Inhalation Powder for Patients with Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Liquidia (NASDAQ: LQDA) has received tentative approval from the FDA for YUTREPIA™ (treprostinil) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The approval indicates that YUTREPIA meets all regulatory standards but must await the expiration of a competing product's exclusivity before final approval. The FDA confirmed that Liquidia's amendment to add PH-ILD to the YUTREPIA NDA was proper. Final approval may occur after May 23, 2025, following the expiration of Tyvaso DPI's 3-year regulatory exclusivity. The tentative approval is based on the Phase 3 INSPIRE trial results, which showed YUTREPIA to be safe and well-tolerated in both treprostinil-naïve patients and those transitioning from nebulized treprostinil.
Liquidia (NASDAQ: LQDA) ha ricevuto l'approvazione preliminare dalla FDA per YUTREPIA™ (polvere per inalazione di treprostinil) per trattare adulti affetti da ipertensione arteriosa polmonare (PAH) e ipertensione polmonare associata a patologie interstiziali polmonari (PH-ILD). L'approvazione indica che YUTREPIA soddisfa tutti gli standard regolatori, ma deve attendere la scadenza dell'esclusività di un prodotto concorrente prima dell'approvazione finale. La FDA ha confermato che la modifica di Liquidia per aggiungere PH-ILD alla NDA di YUTREPIA era appropriata. L'approvazione finale potrebbe avvenire dopo il 23 maggio 2025, a seguito della scadenza dell'esclusività regolatoria triennale di Tyvaso DPI. L'approvazione preliminare si basa sui risultati della fase 3 dello studio INSPIRE, che ha dimostrato che YUTREPIA è sicura e ben tollerata sia in pazienti naive al treprostinil che in quelli che passano da treprostinil nebulizzato.
Liquidia (NASDAQ: LQDA) ha recibido una aprobación preliminar de la FDA para YUTREPIA™ (polvo de inhalación de treprostinil) para tratar a adultos con hipertensión arterial pulmonar (PAH) y hipertensión pulmonar asociada con enfermedades intersticiales pulmonares (PH-ILD). La aprobación indica que YUTREPIA cumple con todos los estándares regulatorios, pero debe esperar a la expiración de la exclusividad de un producto competidor antes de la aprobación final. La FDA confirmó que la enmienda de Liquidia para agregar PH-ILD a la NDA de YUTREPIA era adecuada. La aprobación final puede ocurrir después del 23 de mayo de 2025, tras la expiración de la exclusividad regulatoria de 3 años de Tyvaso DPI. La aprobación preliminar se basa en los resultados del ensayo Fase 3 INSPIRE, que mostró que YUTREPIA es segura y bien tolerada tanto en pacientes naïve a treprostinil como en aquellos que transicionan desde treprostinil nebulizado.
Liquidia (NASDAQ: LQDA)는 성인 환자의 폐동맥 고혈압(PAH) 및 간질성 폐 질환 관련 폐 고혈압(PH-ILD) 치료를 위한 YUTREPIA™ (트레포스틴일) 흡입 분말에 대해 FDA로부터 잠정 승인을 받았습니다. 이 승인은 YUTREPIA가 모든 규제 기준을 충족함을 나타내지만 최종 승인이 내려지기 전에 경쟁 제품의 독점권 만료를 기다려야 합니다. FDA는 YUTREPIA NDA에 PH-ILD를 추가하기 위한 Liquidia의 수정이 적절하다고 확인했습니다. 최종 승인은 2025년 5월 23일 이후에 발생할 수 있습니다, 이는 Tyvaso DPI의 3년간의 규제 독점권 만료 이후입니다. 잠정 승인은 Phase 3 INSPIRE 시험 결과를 기준으로 하며, 이 시험에서 YUTREPIA는 트레포스틴일에 대해 naïve인 환자와 네불라이즈된 트레포스틴일에서 전환하는 환자 모두에게 안전하고 잘 견디는 것으로 나타났습니다.
Liquidia (NASDAQ: LQDA) a reçu une approbation provisoire de la FDA pour YUTREPIA™ (poudre d'inhalation de treprostinil) afin de traiter les adultes atteints de hypertension artérielle pulmonaire (PAH) et hypertension pulmonaire associée à une maladie interstitielle pulmonaire (PH-ILD). L'approbation indique que YUTREPIA répond à toutes les normes réglementaires, mais doit attendre l'expiration de l'exclusivité d'un produit concurrent avant l'approbation finale. La FDA a confirmé que la modification de Liquidia visant à ajouter PH-ILD au NDA de YUTREPIA était appropriée. L'approbation finale pourrait intervenir après le 23 mai 2025, après l'expiration de l'exclusivité réglementaire de trois ans de Tyvaso DPI. L'approbation provisoire est basée sur les résultats de l'essai INSPIRE de phase 3, qui a montré que YUTREPIA est sûr et bien toléré tant chez les patients naïfs au treprostinil que chez ceux qui passent d'un treprostinil nébulisé.
Liquidia (NASDAQ: LQDA) hat von der FDA vorläufige Genehmigung für YUTREPIA™ (Treprostinil-Inhalationspulver) zur Behandlung von Erwachsenen mit pulmonaler arterieller Hypertonie (PAH) und pulmonaler Hypertonie im Zusammenhang mit interstitiellen Lungenerkrankungen (PH-ILD) erhalten. Die Genehmigung zeigt, dass YUTREPIA alle regulatorischen Standards erfüllt, jedoch auf das Auslaufen der Exklusivität eines konkurrierenden Produkts warten muss, bevor die endgültige Genehmigung erteilt wird. Die FDA bestätigte, dass Liquidias Änderung zur Hinzufügung von PH-ILD zur YUTREPIA NDA angemessen war. Die endgültige Genehmigung könnte nach dem 23. Mai 2025 erfolgen, nachdem die dreijährige regulatorische Exklusivität von Tyvaso DPI abgelaufen ist. Die vorläufige Genehmigung basiert auf den Ergebnissen der Phase-3-INSPIRE-Studie, die zeigte, dass YUTREPIA bei sowohl naiven Patienten als auch Patienten, die von nebulisiertem Treprostinil umsteigen, sicher und gut verträglich ist.
- Tentative FDA approval for YUTREPIA to treat both PAH and PH-ILD
- Confirmation that the NDA amendment to add PH-ILD was proper
- YUTREPIA meets all regulatory standards for quality, safety, and efficacy
- Positive results from Phase 3 INSPIRE trial showing safety and tolerability
- Manufacturing, testing, and supply chain meet regulatory standards for quality and safety
- Final approval delayed until May 23, 2025, due to competing product's regulatory exclusivity
- Potential legal challenge against FDA's grant of regulatory exclusivity to United Therapeutics
Insights
The FDA's tentative approval of YUTREPIA for both PAH and PH-ILD is a significant milestone for Liquidia. This decision validates the drug's safety and efficacy profile, potentially expanding treatment options for patients with these rare cardiopulmonary diseases. The inclusion of PH-ILD is particularly noteworthy, as it broadens the drug's applicability. However, the 3-year regulatory exclusivity granted to Tyvaso DPI poses a substantial market entry barrier, delaying YUTREPIA's final approval until May 2025. This setback could impact Liquidia's competitive position and near-term revenue prospects. The company's plan to challenge this exclusivity decision will be important to watch, as it could significantly affect the timeline for YUTREPIA's market entry and patient access.
This news presents a mixed financial outlook for Liquidia (NASDAQ: LQDA). The tentative FDA approval is a positive signal, potentially enhancing investor confidence in the company's pipeline. However, the delayed market entry due to regulatory exclusivity could significantly impact Liquidia's revenue projections and cash flow. The company may need to allocate additional resources for legal challenges, potentially straining its financial position. Investors should closely monitor Liquidia's cash burn rate and any potential need for additional funding to bridge the gap until YUTREPIA's final approval. The stock price might experience volatility as the market reassesses the company's near-term prospects in light of this regulatory hurdle.
The FDA's decision presents a complex legal scenario for Liquidia. The company's plan to challenge the broad grant of regulatory exclusivity to United Therapeutics for Tyvaso DPI will be a critical legal battle. This challenge could set an important precedent in the interpretation of regulatory exclusivity for new dosage forms in the pharmaceutical industry. The outcome will not only affect YUTREPIA's market entry but could also impact future drug approvals and market competition. Liquidia's legal strategy and the strength of their arguments against the FDA's decision will be crucial. This situation highlights the intricate balance between protecting innovation through exclusivity and ensuring patient access to new treatments, a recurring theme in pharmaceutical patent law.
- FDA confirmed that the amendment to add PH-ILD to the YUTREPIA NDA was proper and that application otherwise meets the requirements for approval under the Federal Food, Drug, and Cosmetic Act
- Final Approval of YUTREPIA for PAH and PH-ILD may occur after expiration of 3-year regulatory exclusivity for Tyvaso DPI on May 23, 2025
- FDA’s tentative approval is based upon all information submitted in the NDA, including the status of good manufacturing practices of the facilities used in the manufacture and testing of YUTREPIA
MORRISVILLE, N.C., Aug. 19, 2024 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, announced today that the U.S. Food and Drug Administration (FDA) has granted tentative approval of YUTREPIA™ (treprostinil) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Tentative approval indicates that YUTREPIA has met all regulatory standards for quality, safety and efficacy required for approval in the United States but must await the expiration of regulatory exclusivity of a competing product before final approval can be granted.
Dr. Roger Jeffs, Ph.D., Chief Executive Officer of Liquidia, said: “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 three-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.”
The tentative approval of YUTREPIA is based on findings from the Phase 3 INSPIRE trial which evaluated 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. Results from the INSPIRE study were published in the Pulmonary Circulation Journal in 2022 and the Vascular Pharmacology Journal in 2021. The FDA’s approval also confirms that the supporting information related to the manufacturing, testing and supply chain of YUTREPIA meets regulatory standards for quality and safety in accordance with Good Manufacturing Practices (GMP).
Liquidia remains committed to addressing the unmet needs of PAH and PH-ILD patients and will seek final approval of YUTREPIA as early as possible.
About Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension (PAH) is a rare, chronic, progressive disease caused by hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death. Currently, an estimated 45,000 patients are diagnosed and treated in the United States. There is currently no cure for PAH, so the goals of existing treatments are to alleviate symptoms, maintain or improve functional class, delay disease progression, and improve quality of life.
About Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Pulmonary hypertension (PH) associated with interstitial lung disease (ILD) includes a diverse collection of up to 150 different pulmonary diseases, including interstitial pulmonary fibrosis, chronic hypersensitivity pneumonitis, connective tissue disease related ILD, and chronic pulmonary fibrosis with emphysema (CPFE) among others. Any level of PH in ILD patients is associated with poor 3-year survival. A current estimate of PH-ILD prevalence in the United States is greater than 60,000 patients, though population size in many of these underlying ILD diseases is not yet known due to factors including underdiagnosis and lack of approved treatments until March 2021, when inhaled treprostinil was first approved for this indication.
About YUTREPIA™ (treprostinil) Inhalation Powder
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. Liquidia has completed INSPIRE, or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, an open-label, multi-center phase 3 clinical study of YUTREPIA in patients diagnosed with PAH who are naïve to inhaled treprostinil or who are transitioning from Tyvaso® (nebulized treprostinil). YUTREPIA is currently being studied in the ASCENT trial, an Open-Label Prospective Multicenter Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in Pulmonary Hypertension, with the objective of informing YUTREPIA’s dosing and tolerability profile in patients with PH-ILD. YUTREPIA was previously referred to as LIQ861 in investigational studies.
About Liquidia Corporation
Liquidia Corporation is a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease. The company’s current focus spans the development and commercialization of products in pulmonary hypertension and other applications of its proprietary PRINT® Technology. PRINT enabled the creation of Liquidia’s lead candidate, YUTREPIA™ (treprostinil) inhalation powder, an investigational drug for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The company is also developing L606, an investigational sustained-release formulation of treprostinil administered twice-daily with a next-generation nebulizer, and currently markets generic Treprostinil Injection for the treatment of PAH. To learn more about Liquidia, please visit www.liquidia.com.
Tyvaso® is a registered trademark of United Therapeutics Corporation.
Cautionary Statements Regarding Forward-Looking Statements
This press release may include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release other than statements of historical facts, including statements regarding our future results of operations and financial position, our strategic and financial initiatives, our business strategy and plans and our objectives for future operations, are forward-looking statements. Such forward-looking statements, including statements regarding clinical trials, clinical studies and other clinical work (including the funding therefor, anticipated patient enrollment, safety data, study data, trial outcomes, timing or associated costs), regulatory applications and related submission contents and timelines, including the potential for final FDA approval of the NDA for YUTREPIA, the timeline or outcome related to patent litigation in the U.S. District Court for the District of Delaware or inter partes review proceedings conducted at the PTAB or other litigation instituted by United Therapeutics or others, including rehearings or appeals of decisions in any such proceedings, the issuance of patents by the USPTO and our ability to execute on our strategic or financial initiatives, involve significant risks and uncertainties and actual results could differ materially from those expressed or implied herein. The favorable decisions of courts or other tribunals are not determinative of the outcome of the appeals or rehearings of the decisions. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our financial condition, results of operations, business strategy, short-term and long-term business operations and objectives and financial needs. These forward-looking statements are subject to a number of risks discussed in our filings with the SEC, as well as a number of uncertainties and assumptions. Moreover, we operate in a very competitive and rapidly changing environment and our industry has inherent risks. New risks emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements we may make. In light of these risks, uncertainties and assumptions, the future events discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Nothing in this press release should be regarded as a representation by any person that these goals will be achieved, and we undertake no duty to update our goals or to update or alter any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact Information
Investors:
Jason Adair
919.328.4350
jason.adair@liquidia.com
Media:
Patrick Wallace
919.328.4383
patrick.wallace@liquidia.com
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