FDA Declines to Approve Vanda's Marketing Application for Tradipitant in Gastroparesis
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) announced that the FDA has declined to approve its New Drug Application (NDA) for tradipitant, a treatment for gastroparesis symptoms. The FDA provided a Complete Response Letter (CRL) on September 18, 2024, 185 days after the submission deadline. Vanda argues that the FDA's action fails to meet the Food Drug and Cosmetic Act requirements and disregards the evidence provided, including two placebo-controlled studies and real-world patient experiences. Despite this setback, Vanda believes tradipitant meets the substantial evidence of efficacy standard and plans to continue pursuing marketing authorization. The company will also maintain its expanded access program for gastroparesis patients and plans to submit a separate NDA for tradipitant in motion sickness prevention later this year.
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) ha annunciato che la FDA ha rifiutato di approvare la sua Domanda di Nuovo Farmaco (NDA) per il tradipitant, un trattamento per i sintomi della gastroparesi. La FDA ha fornito una Lettera di Risposta Completa (CRL) il 18 settembre 2024, 185 giorni dopo la scadenza per la presentazione. Vanda sostiene che l'azione della FDA non soddisfa i requisiti previsti dal Food Drug and Cosmetic Act e ignora le prove fornite, inclusi due studi controllati con placebo e le esperienze dei pazienti nel mondo reale. Nonostante questo contrattempo, Vanda crede che il tradipitant soddisfi lo standard di prova di efficacia sostanziale e prevede di continuare a perseguire l’autorizzazione alla commercializzazione. L'azienda manterrà anche il suo programma di accesso ampliato per i pazienti con gastroparesi e prevede di presentare una NDA separata per il tradipitant per la prevenzione della cinetosi entro la fine dell'anno.
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) anunció que la FDA ha rechazado su Solicitud de Nuevo Medicamento (NDA) para el tradipitant, un tratamiento para los síntomas de la gastroparesia. La FDA proporcionó una Carta de Respuesta Completa (CRL) el 18 de septiembre de 2024, 185 días después de la fecha límite de presentación. Vanda argumenta que la acción de la FDA no cumple con los requisitos de la Ley de Alimentos, Medicamentos y Cosméticos y desestima la evidencia presentada, incluyendo dos estudios controlados con placebo y las experiencias de pacientes en el mundo real. A pesar de este contratiempo, Vanda cree que el tradipitant cumple con el estándar de evidencia sustancial de eficacia y planea seguir buscando la autorización de comercialización. La compañía también mantendrá su programa de acceso expandido para pacientes con gastroparesia y planea presentar una NDA separada para el tradipitant en la prevención del mareo por movimiento más adelante este año.
반다 제약(Vanda Pharmaceuticals Inc.)(Nasdaq: VNDA)은 FDA가 위장마비 증상 치료제인 트라디피탄(tradipitant)의 新약 신청(NDA)을 승인하지 않기로 했다고 발표했습니다. FDA는 2024년 9월 18일에 제출 마감일로부터 185일 후에 완전 응답서(CRL)를 제공했습니다. 반다는 FDA의 조치가 식품의약품화장품법의 요건을 충족하지 않으며, 두 개의 위약 대조 연구 및 실제 환자 경험을 포함하여 제출한 증거를 무시했다고 주장하고 있습니다. 이러한 좌절에도 불구하고, 반다는 트라디피탄이 효과의 실질적 증거 기준을 충족한다고 믿으며, 마케팅 허가를 계속 추진할 계획입니다. 또한 이 회사는 위장마비 환자를 위한 확대 접근 프로그램을 유지하고, 올해 말에 운동병 예방을 위한 트라디피탄의 별도의 NDA를 제출할 계획입니다.
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) a annoncé que la FDA a refusé d'approuver sa Demande de Nouveau Médicament (NDA) pour le tradipitant, un traitement des symptômes de la gastroparesie. La FDA a fourni une Lettre de Réponse Complète (CRL) le 18 septembre 2024, 185 jours après la date limite de soumission. Vanda soutient que l'action de la FDA ne répond pas aux exigences de la Loi sur les aliments, les médicaments et les cosmétiques et ignore les preuves fournies, notamment deux études contrôlées par placebo et les expériences des patients dans le monde réel. Malgré ce revers, Vanda estime que le tradipitant répond à la norme d'évidence substantielle d'efficacité et prévoit de continuer à poursuivre l'autorisation de mise sur le marché. L'entreprise maintiendra également son programme d'accès élargi pour les patients atteints de gastroparesie et prévoit de soumettre une NDA séparée pour le tradipitant dans le cadre de la prévention du mal des transports plus tard cette année.
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) gab bekannt, dass die FDA ihren Antrag auf Zulassung eines neuen Medikaments (NDA) für Tradipitant, eine Behandlung von Symptomen der Gastroparese, abgelehnt hat. Die FDA stellte am 18. September 2024 ein Schreiben zur vollständigen Antwort (CRL) aus, 185 Tage nach der Frist für die Einreichung. Vanda argumentiert, dass die Maßnahme der FDA die Anforderungen des Food Drug and Cosmetic Act nicht erfüllt und die vorgelegten Beweise, einschließlich zweier placebo-kontrollierter Studien und realer Patientenerfahrungen, ignoriert. Trotz dieses Rückschlags ist Vanda der Meinung, dass Tradipitant dem Standard für substanzielle Wirksamkeitsnachweise entspricht und plant, die Marktzulassung weiter zu verfolgen. Das Unternehmen wird auch sein erweitertes Zugangsprogramm für Patienten mit Gastroparese aufrechterhalten und plant, noch in diesem Jahr einen separaten NDA-Antrag für Tradipitant zur Prävention von Reisekrankheit einzureichen.
- Vanda plans to submit a separate NDA for tradipitant in motion sickness prevention
- The company is maintaining its expanded access program for gastroparesis patients
- FDA declined to approve Vanda's New Drug Application for tradipitant in gastroparesis treatment
- FDA's response was delayed by more than 185 days, potentially violating regulatory requirements
- FDA suggested additional studies with design and duration inconsistent with expert advice
- FDA refused to convene an expert advisory committee to review the application
The FDA's rejection of Vanda's NDA for tradipitant is a significant setback for the company's gastroparesis program. This decision could have substantial financial implications for Vanda, as gastroparesis represents a large, underserved market with no new FDA-approved treatments in over 40 years.
The FDA's request for additional studies with different designs suggests potential concerns about the robustness of the existing data. This could lead to increased R&D costs and delayed market entry, impacting Vanda's revenue projections and competitive positioning in the gastroparesis space.
Investors should closely monitor Vanda's next steps, including potential appeals, regulatory meetings, or new clinical trials. The company's ability to navigate this setback and advance its pipeline will be important for its long-term growth prospects.
The FDA's Complete Response Letter (CRL) for tradipitant raises significant regulatory concerns. Vanda's claim that the FDA disregarded evidence and delayed action beyond statutory timelines could potentially lead to legal challenges or formal disputes with the agency.
The FDA's reluctance to convene an advisory committee, despite Vanda's requests, is unusual for a novel treatment in an area of high unmet need. This suggests possible deeper issues with the application or data interpretation that weren't resolved during the review process.
Vanda's strategy to continue the expanded access program and pursue a separate NDA for motion sickness indicates a multi-pronged approach to maintain momentum. However, the regulatory path forward for gastroparesis remains uncertain and potentially lengthy.
The FDA's decision is a disappointment for gastroparesis patients, who have treatment options. Tradipitant's potential as a novel therapy for this debilitating condition is significant, especially given the recent association with GLP1 inhibitors, a widely used class of diabetes medications.
The FDA's request for studies with different designs raises questions about the appropriateness of current clinical trial standards for gastroparesis. This highlights the need for better alignment between regulatory requirements and clinical understanding of the disease.
The continued expanded access program is crucial, as it provides real-world data and maintains treatment access for patients. This data could potentially support future regulatory submissions or help refine study designs to address FDA concerns.
On September 18, 2024, the
Gastroparesis is a serious condition that is characterized by delayed gastric emptying and associated with severe nausea, vomiting, difficulty finishing a normal meal and other symptoms that have a significant impact in people's everyday living and functioning. Gastroparesis is often associated with diabetes but is also found in nondiabetic individuals and has recently been associated with the class of GLP1 inhibitors. There has been no effective treatment approved by the FDA in over 40 years.
Vanda's tradipitant application included evidence from two placebo-controlled studies, the results of which were published in peer review journals.1,2 The evidence of efficacy was further supported by exposure response data from a large open label study as well as the real world experience of dozens of patients treated in an expanded access program, some for several years.
The CRL was conclusory in nature, generally disregarded the evidence provided and instead suggested that Vanda conduct additional studies with a design and duration inconsistent with the advice of key experts in the field and not appropriate based on the scientific understanding and natural course of the disorder.
Furthermore, the FDA's action was delayed by more than 185 days and fails to satisfy the requirements specified by the Food Drug and Cosmetic Act (FDCA). The FDCA requires that the FDA review a new drug application and within 180 days of submission provide either an approval or an opportunity for a hearing. In this case the FDA failed to do either.
Despite this disappointing action by the FDA, Vanda believes that the tradipitant application has met the substantial evidence of efficacy standard with a favorable benefit risk profile for the treatment of patients with gastroparesis. While Vanda has repeatedly requested that the FDA convene an expert advisory committee to review the application and advise the Commissioner on the approvability of this application, the FDA has refused to do so. A number of patients currently treated with tradipitant have filed a Citizen Petition urging FDA to approve tradipitant for the treatment of gastroparesis.
Vanda will continue to pursue the marketing authorization for tradipitant and will continue to support the expanded access program that is currently serving several dozen patients with gastroparesis. Vanda encourages any patients with questions about expanded access to contact us at the following email address ExpandedAccess@vandapharma.com.
Vanda plans to submit a separate NDA for tradipitant for the prevention of vomiting in motion sickness later this year.
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on X @vandapharma.
References
- Carlin, J. L., Polymeropoulos, C., Camilleri, M., Lembo, A., Fisher, M., Kupersmith, C., Madonick, D., Moszczynski, P., Smieszek, S., Xiao, C., Birznieks, G., & Polymeropoulos, M. H. (2024). The efficacy of tradipitant in patients with diabetic and idiopathic gastroparesis in phase III randomized placebo-controlled clinical trial. Clinical Gastroenterology and Hepatology. Available online: https://www.cghjournal.org/article/S1542-3565(24)00050-8/fulltext
- Carlin, J. L., Lieberman, V. R., Dahal, A., Keefe, M. S., Xiao, C., Birznieks, G., Abell, T. L., Lembo, A., Parkman, H. P., & Polymeropoulos, M. H. (2021). Efficacy and Safety of Tradipitant in Patients With Diabetic and Idiopathic Gastroparesis in a Randomized, Placebo-Controlled Trial. Gastroenterology, 160(1), 76–87.e4. Available online: https://doi.org/10.1053/j.gastro.2020.07.029
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this press release, including, but not limited to, statements regarding Vanda's plans for pursuit of FDA approval of tradipitant for the treatment of symptoms of gastroparesis and motion sickness are "forward-looking statements" under the securities laws. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Forward-looking statements are based upon current expectations and assumptions that involve risks, changes in circumstances and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, Vanda's ability to complete and submit to the FDA the NDA for tradipitant for the treatment of motion sickness within the specified timeframe and the FDA's assessment of the sufficiency of the data packages to be included in the NDA for tradipitant. Therefore, no assurance can be given that the results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's most recent Annual Report on Form 10-K, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the
All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Corporate Contact:
Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
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Collected Strategies
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FAQ
What was the FDA's decision on Vanda's tradipitant for gastroparesis?
What evidence did Vanda provide in its tradipitant application?
How did Vanda respond to the FDA's decision on tradipitant (VNDA)?
What are Vanda's future plans for tradipitant after the FDA rejection?
