Vanda's Letter to FDA Commissioner Highlights Faulty Gastroparesis NDA Review
Vanda Pharmaceuticals (NASDAQ: VNDA) has issued a letter to FDA Commissioner Robert M. Califf addressing concerns over the agency's rejection of its New Drug Application (NDA) for tradipitant, a treatment for gastroparesis symptoms. The letter follows a Complete Response Letter (CRL) received on September 18, 2024, which denied approval for the drug.
The company criticizes the FDA's review process, highlighting several issues including: the lack of response from Dr. Nikolov to their concerns about the CRL, the denial of their request for an Advisory Committee meeting, and what they describe as a 'disturbing pattern of conduct' at the FDA. The letter points out a significant decline in Advisory Committee meetings, from 55% of drug applications in 2010 to 6% in 2021.
Vanda argues that the FDA's current policies and practices need realignment with scientific evidence and law, particularly questioning the Commissioner's stance on only overruling civil servants' decisions in cases of 'corruption' or 'temporary insanity.'
Vanda Pharmaceuticals (NASDAQ: VNDA) ha inviato una lettera al Commissario della FDA Robert M. Califf esprimendo preoccupazioni riguardo il rifiuto dell'agenzia della sua Domanda di Nuovo Farmaco (NDA) per tradipitant, un trattamento per i sintomi della gastroparesi. La lettera segue una Lettera di Risposta Completa (CRL) ricevuta il 18 settembre 2024, che ha negato l'approvazione del farmaco.
L'azienda critica il processo di revisione della FDA, evidenziando diversi problemi tra cui: la mancanza di risposta da parte del Dr. Nikolov alle loro preoccupazioni riguardo alla CRL, il rifiuto della loro richiesta per una riunione del Comitato Consultivo e quella che definiscono un 'disturbante modello di condotta' da parte della FDA. La lettera sottolinea un significativo calo delle riunioni del Comitato Consultivo, passando dal 55% delle domande di farmaci nel 2010 al 6% nel 2021.
Vanda sostiene che le attuali politiche e pratiche della FDA necessitano di un allineamento reale con le evidenze scientifiche e la legge, ponendo in particolare interrogativi sulla posizione del Commissario di sovvertire le decisioni dei funzionari pubblici solo in casi di 'corruzione' o 'tempesta temporanea di follia.'
Vanda Pharmaceuticals (NASDAQ: VNDA) ha emitido una carta al Comisionado de la FDA, Robert M. Califf, abordando preocupaciones sobre el rechazo de la agencia a su Solicitud de Nuevo Medicamento (NDA) para tradipitant, un tratamiento para los síntomas de la gastroparesia. La carta sigue a una Carta de Respuesta Completa (CRL) recibida el 18 de septiembre de 2024, que negó la aprobación del medicamento.
La empresa critica el proceso de revisión de la FDA, destacando varios problemas, incluyendo: la falta de respuesta del Dr. Nikolov a sus preocupaciones sobre la CRL, el rechazo de su solicitud para una reunión del Comité Asesor y lo que describen como un 'patrón perturbador de conducta' en la FDA. La carta señala una disminución significativa en las reuniones del Comité Asesor, del 55% de las solicitudes de medicamentos en 2010 al 6% en 2021.
Vanda argumenta que las políticas y prácticas actuales de la FDA necesitan ser realineadas con la evidencia científica y la ley, cuestionando en particular la postura del Comisionado de anular las decisiones de los funcionarios públicos solo en casos de 'corrupción' o 'locura temporal.'
반다 제약 (NASDAQ: VNDA)는 FDA 위원장인 로버트 M. 캘리프에게 편지를 보내 위원회의 전통팟안트에 대한 신약신청(NDA) 거부에 대한 우려를 전했습니다. 이 편지는 2024년 9월 18일에 수신된 완전 응답서(CRL) 이후 작성된 것으로, 약물의 승인을 거부한 내용입니다.
회사는 FDA의 리뷰 과정에 대해 비판하며, 다음과 같은 여러 문제를 강조합니다: CRL에 대한 그들의 우려에 대한 닥터 니콜로프의 응답 부족, 자문 위원회 회의 요청 거부, 그리고 그들이 '불안한 행동 패턴'으로 묘사하는 FDA의 행위입니다. 이 편지는 2010년 55%에서 2021년 6%로 줄어든 자문 위원회 회의의 감소를 강조합니다.
반다는 FDA의 현재 정책과 관행이 과학적 증거 및 법률에 따라 실제 재정렬이 필요하다고 주장하며, 특히 위원장이 '부패' 또는 '일시적인 광기'의 경우에만 공무원의 결정을 번복하는 것에 대한 입장을 의문시합니다.
Vanda Pharmaceuticals (NASDAQ: VNDA) a adressé une lettre au commissaire de la FDA, Robert M. Califf, soulevant des inquiétudes concernant le refus de l'agence de sa demande de médicament nouveau (NDA) pour le tradipitant, un traitement des symptômes de la gastroparesie. La lettre fait suite à une lettre de réponse complète (CRL) reçue le 18 septembre 2024, qui a refusé l'approbation du médicament.
L'entreprise critique le processus d'examen de la FDA, mettant en avant plusieurs problèmes, notamment : le manque de réponse du Dr. Nikolov à leurs préoccupations concernant la CRL, le rejet de leur demande de réunion du comité consultatif et ce qu'ils qualifient de 'modèle de conduite perturbant' à la FDA. La lettre souligne une baisse significative des réunions du comité consultatif, passant de 55 % des demandes de médicaments en 2010 à 6 % en 2021.
Vanda soutient que les politiques et pratiques actuelles de la FDA doivent être réalignées avec les preuves scientifiques et la loi, remettant en question la position du commissaire sur l'annulation des décisions des fonctionnaires publics uniquement dans des cas de 'corruption' ou de 'folie temporaire.'
Vanda Pharmaceuticals (NASDAQ: VNDA) hat einen Brief an den FDA-Kommissar Robert M. Califf geschrieben, in dem Bedenken über die Ablehnung des Antrags auf neue Arzneimittel (NDA) für Tradipitant, ein Mittel gegen Symptome der Gastroparese, geäußert werden. Der Brief folgt auf ein Complete Response Letter (CRL), das am 18. September 2024 erhalten wurde, und die Genehmigung des Medikaments verweigerte.
Das Unternehmen kritisiert den Überprüfungsprozess der FDA und hebt mehrere Probleme hervor, darunter: die fehlende Antwort von Dr. Nikolov auf ihre Bedenken bezüglich des CRL, die Ablehnung ihrer Anfrage für eine Sitzung des Expertenausschusses und was sie als ein 'beunruhigendes Verhaltensmuster' der FDA beschreiben. Der Brief weist auf einen signifikanten Rückgang der Sitzungen des Expertenausschusses hin, von 55 % der Arzneimittelanträge im Jahr 2010 auf 6 % im Jahr 2021.
Vanda argumentiert, dass die aktuellen Richtlinien und Praktiken der FDA mit wissenschaftlichen Erkenntnissen und dem Gesetz in Einklang gebracht werden müssen, insbesondere wird die Position des Kommissars in Frage gestellt, nur die Entscheidungen von Beamten in Fällen von 'Korruption' oder 'vorübergehender Geisteskrankheit' außer Kraft zu setzen.
- None.
- FDA rejected Vanda's NDA for tradipitant in gastroparesis treatment
- FDA denied company's request for Advisory Committee meeting
- No response received from FDA regarding concerns about the Complete Response Letter
- Regulatory setback delays potential market entry for tradipitant
Insights
The public confrontation between Vanda and the FDA Commissioner over the tradipitant NDA rejection represents a significant regulatory dispute with potential implications for both the company and broader industry practices. The key issues center on FDA's declining use of Advisory Committees (from
The confrontational tone of Vanda's letter, directly challenging the FDA Commissioner's leadership approach and decision-making framework, could potentially strain future regulatory interactions. The company's inability to secure an Advisory Committee review for tradipitant, combined with the apparent lack of response to their scientific arguments, suggests a challenging path forward for the gastroparesis treatment's approval.
This dispute highlights broader industry concerns about FDA's current review processes and could influence how other pharmaceutical companies approach their regulatory strategies. The criticism of FDA's reduced reliance on Advisory Committees may pressure the agency to reconsider its current practices, particularly for controversial or complex applications.
Vanda's aggressive stance against the FDA's review process reveals deep concerns about their gastroparesis drug's commercial prospects. For a small-cap company (
The lack of response from FDA officials and denial of an Advisory Committee review suggests a potentially prolonged regulatory process ahead. This situation typically leads to increased R&D costs and delayed market entry, particularly challenging for smaller pharmaceutical companies with resources. The market implications are concerning, as extended regulatory disputes often lead to increased cash burn and potential need for additional capital raising.
As previously reported, Vanda has sought approval from the
Today, Vanda issued a letter to FDA Commissioner Robert M. Califf, MD, regarding the September 18, 2024 CRL in reference to Vanda's NDA for tradipitant for the treatment of gastroparesis. The full letter is shown below:
_____________________________________________________________________
Dear Dr. Califf:
We are writing to bring your attention to a disturbing pattern of conduct at FDA that impairs the credibility of the agency and harms the American public. In an interview last year you stated that you would not overrule decisions made by civil servants at the Agency except in certain cases of "corruption" or "temporary insanity" of the decision maker.1 Neither the public nor regulated entities like Vanda are able to determine what instances of "corruption" or "temporary insanity" would in your view merit overruling lower-level FDA employee decisions. This opacity in decisionmaking and oversight has allowed a culture of obfuscation and closemindedness to fester at FDA. And your agency's review of our application to market tradipitant is no exception.
Three months ago, Vanda received a complete response letter (CRL), dated September 18, 2024, in response to the new drug application (NDA) Vanda submitted on September 18, 2023, for the use of tradipitant for the treatment of symptoms of gastroparesis, a serious and debilitating gastrointestinal disorder. We wrote to Dr. Nikolov expressing our surprise "by the sheer disregard for the facts, evidence, and basic scientific principles contained in the complete response"—a letter that could not "possibly reflect a legitimate regulatory review" as it did not "provide reasoned explanations or engage with the evidence we presented on its merit—including voluminous evidence from experts."
Three months later, Dr. Nikolov has not even acknowledged that letter, let alone provided a response. This is unacceptable, and stems from the seriously misguided nature of your position that you will not overrule decisions made by civil servants at the Agency except in extreme situations such as "corruption" or "temporary insanity" of the decision maker. As a political appointee and head of the agency, you are the only person accountable to the American public—a public that needs to know that the agency will follow the law and ensure courteous and civil behavior by agency employees.
In addition, FDA has denied our request to convene an Advisory Committee to consider our application for tradipitant, a process by which experts and the public can voice their opinion on our application. As you are no doubt aware, the number of Advisory Committee meetings convened by the agency has drastically declined over the last few years. See Cheri Banks, The Future of Voting for FDA Advisory Committees, Federation of American Scientists (Sept. 9, 2024), perma.cc/L8R3-2FJD (noting that in 2021,
I understand that you may be leaving the agency in the new administration, but I hope that you will consider this letter, and I would welcome your thoughts in response. FDA's policies, practices, and culture must be evaluated and corrected so as to align with scientific evidence and the law.
Sincerely,
Mihael H. Polymeropoulos, M.D.
Chief Executive Officer
Vanda Pharmaceuticals Inc.
1. MedPage Today, Politics and Controversy in the FDA, YouTube (Apr. 11, 2023) (statement of Commissioner Robert Califf, M.D.), available at https://www.youtube.com/watch?reload=9&app=desktop&v=zGrnzM51Jrs.
______________________________________________________________________
The text of the letter to Dr. Nikolov, referenced in the second paragraph of the letter above, is available at: https://assets.vandapharma.com/pdfs/vanda-letter-to-dr-nikolov.pdf
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.
Corporate Contact:
Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
Jim Golden / Jack Kelleher / Dan Moore
Collected Strategies
VANDA-CS@collectedstrategies.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/vandas-letter-to-fda-commissioner-highlights-faulty-gastroparesis-nda-review-302346357.html
SOURCE Vanda Pharmaceuticals Inc.
FAQ
Why did the FDA reject Vanda's (VNDA) tradipitant NDA for gastroparesis in September 2024?
What is the significance of FDA denying VNDA's Advisory Committee meeting request for tradipitant?
What are the next steps for Vanda (VNDA) following the tradipitant CRL?
How long has VNDA's tradipitant been under FDA review for gastroparesis?
