Disc Medicine Announces Positive Pre-NDA Meeting and Confirms Plans to Submit NDA for Bitopertin in Erythropoietic Protoporphyria (EPP) in October 2025
Disc Medicine (NASDAQ:IRON) has received positive feedback from its pre-NDA meeting with the FDA regarding bitopertin, its treatment for Erythropoietic Protoporphyria (EPP). The company has confirmed plans to submit its New Drug Application (NDA) in October 2025 under the FDA's accelerated approval pathway.
CEO John Quisel expressed confidence in the alignment with the FDA regarding the submission's timing and content. The company is preparing for its first potential product launch, marking a significant milestone in bringing bitopertin to the EPP community.
Disc Medicine (NASDAQ:IRON) ha ricevuto un riscontro positivo dal suo incontro pre-NDA con la FDA riguardo bitopertin, il suo trattamento per la Porfiria Eritropoietica Protoporfiria (EPP). L'azienda ha confermato l'intenzione di presentare la sua Domanda di Nuovo Farmaco (NDA) entro ottobre 2025 seguendo la procedura di approvazione accelerata della FDA.
Il CEO John Quisel ha espresso fiducia nell'allineamento con la FDA riguardo i tempi e i contenuti della presentazione. L'azienda si sta preparando per il suo primo possibile lancio di prodotto, segnando una tappa importante nel portare bitopertin alla comunità EPP.
Disc Medicine (NASDAQ:IRON) ha recibido comentarios positivos de su reunión previa a la NDA con la FDA sobre bitopertin, su tratamiento para la Porfiria Eritropoyética (EPP). La compañía ha confirmado sus planes de presentar su Solicitud de Nuevo Medicamento (NDA) en octubre de 2025 bajo la vía de aprobación acelerada de la FDA.
El CEO John Quisel expresó confianza en la alineación con la FDA respecto al momento y contenido de la presentación. La empresa se está preparando para su primer posible lanzamiento de producto, marcando un hito importante para llevar bitopertin a la comunidad EPP.
Disc Medicine (NASDAQ:IRON)은(는) 적혈구 형성성 프로토포르피린증(EPP) 치료제인 비토퍼틴(bitopertin)에 대해 FDA와의 NDA 사전 미팅에서 긍정적인 피드백을 받았습니다. 회사는 FDA의 가속 승인 절차에 따라 2025년 10월에 신약 신청서(NDA)를 제출할 계획임을 확인했습니다.
CEO 존 퀴젤은 제출 시기와 내용에 대해 FDA와의 조율에 자신감을 나타냈습니다. 회사는 비토퍼틴을 EPP 환자 커뮤니티에 선보이는 중요한 이정표가 될 첫 제품 출시를 준비하고 있습니다.
Disc Medicine (NASDAQ:IRON) a reçu un retour positif lors de sa réunion pré-NDA avec la FDA concernant bitopertin, son traitement contre la protoporphyrie érythropoïétique (EPP). La société a confirmé son intention de soumettre sa demande de nouveau médicament (NDA) en octobre 2025 dans le cadre de la procédure d'approbation accélérée de la FDA.
Le PDG John Quisel a exprimé sa confiance quant à l'alignement avec la FDA sur le calendrier et le contenu de la soumission. La société se prépare à son premier lancement potentiel de produit, marquant une étape importante pour apporter bitopertin à la communauté EPP.
Disc Medicine (NASDAQ:IRON) hat positives Feedback von seinem Pre-NDA-Meeting mit der FDA bezüglich Bitopertin, seiner Behandlung für erythropoetische Protoporphyrie (EPP), erhalten. Das Unternehmen hat Pläne bestätigt, seinen New Drug Application (NDA) im Oktober 2025 über den beschleunigten Zulassungsweg der FDA einzureichen.
CEO John Quisel zeigte sich zuversichtlich, dass die Abstimmung mit der FDA hinsichtlich des Zeitpunkts und Inhalts der Einreichung gelungen ist. Das Unternehmen bereitet sich auf seine erste mögliche Produkteinführung vor, was einen bedeutenden Meilenstein für die Versorgung der EPP-Gemeinschaft mit Bitopertin darstellt.
- Successful pre-NDA meeting with FDA confirming submission plans
- Accelerated approval pathway eligibility confirmed
- On track for October 2025 NDA submission
- Alignment with FDA on submission content and timing
- Product approval not guaranteed despite positive pre-NDA meeting
- Several months until NDA submission in October 2025
WATERTOWN, Mass., July 21, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced positive feedback from its pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned NDA submission for bitopertin in EPP.
“At the pre-NDA meeting, we confirmed alignment with the agency on the expected timing and content of our NDA submission and, as a result, we are on track for a submission in October of this year,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We appreciate the timely and constructive feedback from the agency as we work to bring bitopertin to the EPP community and prepare our organization for its first potential product launch.”
The purpose of the meeting was to confirm the FDA’s expectations for the proposed timing, format and content of the planned NDA submission. Based on feedback from the FDA, Disc plans to submit an NDA for bitopertin in EPP in October 2025 under the FDA’s accelerated approval pathway based on Disc’s existing data package.
About Bitopertin
Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, and an open-label extension HELIOS trial.
Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021.
About Erythropoietic Protoporphyria (EPP)
Erythropoietic protoporphyria (EPP), including X-linked Protoporphyria (XLP), is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20
About Disc Medicine
Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.
Disc Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to the registrational pathway for bitopertin, including the timing of a potential NDA submission. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2024, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
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