Liquidia Files Litigation to Challenge Regulatory Exclusivity Blocking Access to YUTREPIA™ (treprostinil) inhalation powder for Patients Suffering with PAH and PH-ILD
Liquidia (NASDAQ: LQDA) has filed a lawsuit challenging the FDA's decision to grant 3-year new clinical investigation exclusivity (NCI exclusivity) to Tyvaso DPI®. This exclusivity is delaying the final approval of Liquidia's YUTREPIA™ (treprostinil) inhalation powder for treating pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) until after May 23, 2025.
Liquidia argues that the FDA's decision violates congressional intent, which allows NCI exclusivity only for true innovations supported by new clinical studies demonstrating safety and/or efficacy. The company believes this exclusivity should be vacated, allowing YUTREPIA to enter the market immediately. Separately, United Therapeutics has voluntarily dismissed its complaint against the FDA challenging the acceptance of Liquidia's amended NDA for YUTREPIA.
Liquidia (NASDAQ: LQDA) ha presentato una causa legale contro la decisione della FDA di concedere un'esclusiva per una nuova indagine clinica di 3 anni (esclusiva NCI) per Tyvaso DPI®. Questa esclusiva sta ritardando l'approvazione finale della polvere per inalazione YUTREPIA™ (treprostinil) di Liquidia per il trattamento dell'ipertensione arteriosa polmonare (PAH) e dell'ipertensione polmonare associata a malattia polmonare interstiziale (PH-ILD) fino a dopo il 23 maggio 2025.
Liquidia sostiene che la decisione della FDA viola l'intento del Congresso, che consente l'esclusiva NCI solo per vere innovazioni supportate da nuovi studi clinici che dimostrano sicurezza e/o efficacia. L'azienda crede che questa esclusiva dovrebbe essere annullata, permettendo a YUTREPIA di entrare immediatamente nel mercato. Separatamente, United Therapeutics ha ritirato volontariamente il suo ricorso contro la FDA per contestare l'accettazione della NDA modificata di Liquidia per YUTREPIA.
Liquidia (NASDAQ: LQDA) ha presentado una demanda impugnando la decisión de la FDA de otorgar una exclusividad de nueva investigación clínica de 3 años (exclusividad NCI) para Tyvaso DPI®. Esta exclusividad está retrasando la aprobación final del polvo de inhalación YUTREPIA™ (treprostinil) de Liquidia para el tratamiento de la hipertensión arterial pulmonar (PAH) y la hipertensión pulmonar asociada con enfermedad pulmonar intersticial (PH-ILD) hasta después del 23 de mayo de 2025.
Liquidia argumenta que la decisión de la FDA infringe la intención del Congreso, que permite la exclusividad NCI solo para verdaderas innovaciones respaldadas por nuevos estudios clínicos que demuestren seguridad y/o eficacia. La empresa cree que esta exclusividad debería ser anulada, permitiendo que YUTREPIA ingrese al mercado de inmediato. Por separado, United Therapeutics ha desestimado voluntariamente su queja contra la FDA impugnando la aceptación de la NDA enmendada de Liquidia para YUTREPIA.
Liquidia (NASDAQ: LQDA)는 FDA의 Tyvaso DPI®에 대한 3년 신규 임상 조사 독점(NCI 독점) 부여 결정을 도전하는 소송을 제기했습니다. 이 독점은 Liquidia의 YUTREPIA™ (treprostinil) 흡입 분말이 폐동맥고혈압(PAH) 및 간질성 폐질환(PH-ILD)과 관련된 폐고혈압 치료를 위해 2025년 5월 23일 이후까지 최종 승인을 지연시키고 있습니다.
Liquidia는 FDA의 결정이 새로운 임상 연구를 통해 안전성과/또는 효과를 입증한 진정한 혁신에 대해서만 NCI 독점이 허용된다는 의회의 의도를 위배한다고 주장합니다. 회사는 이 독점이 해제되어 YUTREPIA가 즉시 시장에 진입할 수 있어야 한다고 믿고 있습니다. 별도로 United Therapeutics는 Liquidia의 YUTREPIA에 대한 수정된 NDA 수용에 반대하는 FDA에 대한 고소를 자발적으로 철회했습니다.
Liquidia (NASDAQ: LQDA) a déposé une plainte contestation la décision de la FDA d'accorder une exclusivité de nouvelle investigation clinique de 3 ans (exclusivité NCI) à Tyvaso DPI®. Cette exclusivité retarde l'approbation finale de la poudre d'inhalation YUTREPIA™ (treprostinil) de Liquidia pour traiter l'hypertension artérielle pulmonaire (PAH) et l'hypertension pulmonaire associée à une maladie pulmonaire interstitielle (PH-ILD) jusqu'après le 23 mai 2025.
Liquidia soutient que la décision de la FDA viole l'intention du Congrès, qui ne permet l'exclusivité NCI que pour de vraies innovations soutenues par de nouvelles études cliniques démontrant la sécurité et/ou l'efficacité. L'entreprise estime que cette exclusivité devrait être annulée, permettant à YUTREPIA d'entrer sur le marché immédiatement. D'autre part, United Therapeutics a volontairement retiré sa plainte contre la FDA contestant l'acceptation de la NDA amendée de Liquidia pour YUTREPIA.
Liquidia (NASDAQ: LQDA) hat eine Klage eingereicht, die die Entscheidung der FDA anfechtet, Tyvaso DPI® eine 3-jährige Exklusivität für neue klinische Untersuchungen (NCI-Exklusivität) zu gewähren. Diese Exklusivität verzögert die endgültige Genehmigung von Liquidias YUTREPIA™ (treprostinil) Inhalationspulver zur Behandlung von pulmonaler arterieller Hypertonie (PAH) und pulmonaler Hypertonie bei interstitiellen Lungenerkrankungen (PH-ILD) bis nach dem 23. Mai 2025.
Liquidia argumentiert, dass die Entscheidung der FDA die Absicht des Kongresses verletzt, die NCI-Exklusivität nur für tatsächliche Innovationen zu gewähren, die durch neue klinische Studien belegt werden, die Sicherheit und/oder Wirksamkeit demonstrieren. Das Unternehmen ist der Ansicht, dass diese Exklusivität aufgehoben werden sollte, damit YUTREPIA sofort auf den Markt kommen kann. Separat hat United Therapeutics freiwillig seine Klage gegen die FDA zurückgezogen, in der die Annahme von Liquidias geänderter NDA für YUTREPIA angefochten wurde.
- FDA granted tentative approval of YUTREPIA™ for PAH and PH-ILD treatment
- Liquidia is actively pursuing legal action to potentially expedite YUTREPIA's market entry
- United Therapeutics dismissed its complaint against FDA regarding Liquidia's NDA amendment
- Final approval of YUTREPIA is delayed until after May 23, 2025 due to NCI exclusivity granted to Tyvaso DPI®
- Legal challenges may result in additional costs and uncertain outcomes for Liquidia
Insights
This litigation challenges the FDA's decision to grant 3-year new clinical investigation exclusivity (NCI) to a competitor's product, delaying Liquidia's YUTREPIA™ approval. The key legal argument hinges on the interpretation of NCI exclusivity requirements. Liquidia contends that the FDA's decision violates congressional intent, which aimed to balance innovation incentives with market competition.
The case raises important questions about the scope and application of NCI exclusivity. If Liquidia succeeds, it could set a precedent limiting the FDA's discretion in granting such exclusivity, potentially accelerating market entry for generic or similar drugs. This could have far-reaching implications for the pharmaceutical industry, potentially reducing barriers to market entry and increasing competition in drug markets.
This litigation could significantly impact the pulmonary hypertension treatment market. If Liquidia prevails, YUTREPIA™ could enter the market earlier, potentially disrupting United Therapeutics' market position. This could lead to increased competition and potentially lower prices for patients.
The case highlights the ongoing tension between innovation protection and market access in the pharmaceutical industry. A favorable outcome for Liquidia might encourage other companies to challenge similar exclusivity grants, potentially accelerating the entry of new treatments across various therapeutic areas. Investors should closely monitor this case as it could have broader implications for the pharmaceutical sector's competitive landscape and pricing dynamics.
The dispute over YUTREPIA™'s approval delay underscores the complexities in bringing new formulations of existing drugs to market. Treprostinil, the active ingredient in both YUTREPIA™ and Tyvaso DPI®, is a well-established treatment for pulmonary hypertension. The key innovation here appears to be the delivery method - an inhalation powder.
This case raises questions about what constitutes a significant clinical innovation worthy of exclusivity. If Liquidia's arguments prevail, it could lower the bar for approving alternative formulations of existing drugs. This could potentially accelerate the development of more patient-friendly drug delivery systems, benefiting those with chronic conditions like PAH and PH-ILD who require long-term treatment.
MORRISVILLE, N.C., Aug. 22, 2024 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, announced today that it has filed litigation in the U.S. District Court of the District of Columbia (Case No. 1:24-cv-02428) that challenges the recent decision by the United States Food and Drug Administration (FDA) to grant 3-year new clinical investigation exclusivity (NCI exclusivity) to Tyvaso DPI®. FDA granted tentative approval of YUTREPIA™ (treprostinil) inhalation powder to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) patients on August 16, 2024. As a result of the FDA’s award of NCI exclusivity to Tyvaso DPI, the final approval of YUTREPIA™ (treprostinil) inhalation powder is currently delayed until after the expiration of the exclusivity on May 23, 2025.
Dr. Roger Jeffs, Ph.D., Chief Executive Officer of Liquidia, said: “The FDA’s action improperly allows United Therapeutics to tack on yet another regulatory exclusivity, stifling competition and patient choice. This decision violates clear congressional intent to allow NCI exclusivity only for true innovations that are supported by new clinical studies that demonstrate safety and/or efficacy of the innovation. It is our strong belief that the FDA’s decision to grant Tyvaso DPI this new NCI exclusivity should be vacated, and Liquidia should be allowed to bring YUTREPIA to market for the benefit of patients immediately.”
In establishing NCI exclusivity, Congress sought to encourage innovation in drug development while also ensuring patient access to alternative drugs through competition. Accordingly, NCI exclusivity is only granted for a period of three years from the date of an FDA approval that is supported by certain types of clinical studies, expressly excluding bioavailability studies and clinical investigations that a drug sponsor has previously submitted to FDA. Additionally, NCI exclusivity is limited in scope to the innovative change supported by the new clinical investigation.
Separately, on August 20, 2024, United Therapeutics voluntarily dismissed, without prejudice, the complaint it had filed against the FDA in the U.S. District Court for the District of Columbia, challenging the FDA’s acceptance of Liquidia’s amended NDA for YUTREPIA for review. With the FDA’s grant of tentative approval for YUTREPIA, the FDA decided that it was proper for Liquidia to add PH-ILD to the NDA for YUTREPIA via an amendment.
Liquidia remains committed to addressing the unmet needs of PAH and PH-ILD patients and is seeking final approval of YUTREPIA as soon 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® and Tyvaso DPI® are a registered trademarks 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 of our lawsuit against the FDA, 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 voluntary dismissal of a lawsuit without prejudice allows the underlying claims to be reasserted and does not address the merits of the underlying claims. 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
FAQ
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