Jaguar Health Submits Orphan Drug Designation Application to FDA for Crofelemer for Cancer Therapy-Related Diarrhea (CTD) in Patients with Breast Cancer with Metastasis Identified by FDA as a Distinct Condition
Jaguar Health (NASDAQ:JAGX) has submitted an orphan drug designation (ODD) application to the FDA for crofelemer, targeting diarrhea treatment in breast cancer patients with brain metastasis who are receiving targeted therapy with or without standard chemotherapy.
The application leverages FDA's recognition of brain metastasis as a distinct condition from primary cancers, evidenced by seven ODDs awarded between 2015-2024. If granted, the ODD would provide seven years of marketing exclusivity, tax credits for clinical testing, and filing fee relief.
The company plans to pursue Breakthrough Therapy and/or Fast Track designations to expedite regulatory pathways. Crofelemer already holds ODDs for short bowel syndrome, microvillus inclusion disease, and cholera-related diarrhea.
Jaguar Health (NASDAQ:JAGX) ha presentato una domanda di designazione di farmaco orfano (ODD) alla FDA per crofelemer, mirata al trattamento della diarrea in pazienti con cancro al seno che hanno metastasi cerebrali e che ricevono terapie mirate con o senza chemioterapia standard.
La domanda si basa sul riconoscimento della FDA che le metastasi cerebrali costituiscono una condizione distinta dai tumori primari, come dimostrano sette ODD assegnate tra il 2015 e il 2024. Se concessa, l'ODD offrirebbe sette anni di esclusività di mercato, crediti d'imposta per gli studi clinici e agevolazioni sulle tasse di deposito.
L'azienda intende proseguire con designazioni di Breakthrough Therapy e/o Fast Track per accelerare i percorsi regolatori. Il crofelemer possiede già ODD per la sindrome dell'intestino corto, la malattia da inclusione delle microville, e diarrea associata al colera.
Jaguar Health (NASDAQ:JAGX) ha presentado una solicitud de designación de fármaco huérfano (ODD) ante la FDA para crofelemer, dirigida al tratamiento de la diarrea en pacientes con cáncer de mama que presentan metástasis en el cerebro y que reciben terapias dirigidas con o sin quimioterapia estándar.
La solicitud se apoya en el reconocimiento de la FDA de que las metástasis cerebrales son una condición distinta de los tumores primarios, como lo demuestran siete ODD concedidas entre 2015 y 2024. De ser aprobada, la ODD proporcionaría siete años de exclusividad de comercialización, créditos fiscales para ensayos clínicos y alivio de tarifas de presentación.
La empresa planea perseguir designaciones de Terapia Innovadora y/o Fast Track para acortar las rutas regulatorias. Crofelemer ya posee ODD para síndrome del intestino corto, enfermedad de inclusión de microvilos y diarrea relacionada con el cólera.
Jaguar Health (NASDAQ:JAGX)가 crofelemer에 대한 FDA의 고아의약품 지정(ODD) 신청을 제출했으며, 표적 치료를 받는 유방암 환자 중 뇌 전이 환자에서 표준 화학요법 여부에 관계없이 설사를 치료하기 위한 것을 목표로 한다.
이 신청은 FDA가 뇌 전이를 1차 암과 구별된 상태로 인식한다는 근거를 활용하며, 2015년~2024년 사이에 7건의 ODD가 수여된 사례를 통해 입증된다. 승인될 경우 ODD는 7년의 시장 독점, 임상 시험에 대한 세액공제, 제출 수수료 면제를 제공할 것이다.
회사는 규제 경로를 신속화하기 위해 Breakthrough Therapy 및/또는 Fast Track 지정을 추진할 계획이다. crofelemer은 이미 짧은 창자 증후군, 마이크로빌루스 포함 질환, 콜라라 관련 설사를 위한 ODD를 보유하고 있다.
Jaguar Health (NASDAQ:JAGX) a soumis une demande de désignation de médicament orphelin (ODD) à la FDA pour le crofelemer, destinée au traitement de la diarrhée chez les patientes atteintes d'un cancer du sein avec métastases cérébrales et recevant une thérapie ciblée avec ou sans chimiothérapie standard.
La demande s’appuie sur la reconnaissance par la FDA des métastases cérébrales comme une condition distincte des cancers primitifs, comme en témoignent sept ODD attribuées entre 2015 et 2024. Si elle est accordée, l’ODD offrirait sept ans d’exclusivité marketing, des crédits d’impôt pour les essais cliniques et une exonération des frais de dossier.
L’entreprise prévoit de poursuivre les désignations de Breakthrough Therapy et/ou Fast Track pour accélérer les voies réglementaires. Le crofelemer détient déjà des ODD pour le syndrome de l’intestin court, la maladie d’inclusion microvillulaire et la diarrhée liée au choléra.
Jaguar Health (NASDAQ:JAGX) hat einen Antrag auf Orphan Drug Designation (ODD) bei der FDA für Crofelemer gestellt, der die Behandlung von Durchfall bei Brustkrebspatientinnen mit Hirnmetastasen ins Visier nimmt, die eine zielgerichtete Therapie mit oder ohne Standardchemotherapie erhalten.
Der Antrag stützt sich auf die Anerkennung der FDA, dass Hirnmetastasen eine eigenständige Erkrankung im Vergleich zu Primärtumoren darstellen, was durch sieben zwischen 2015 und 2024 verliehene ODDs belegt wird. Wird er genehmigt, würde die ODD sieben Jahre Marktexklusivität, Steuergutschriften für klinische Tests und Gebührenbefreiungen bei der Einreichung bieten.
Das Unternehmen plant, Designationen wie Breakthrough Therapy und/oder Fast Track zu verfolgen, um regulatorische Wege zu beschleunigen. Crofelemer besitzt bereits ODDs für das Kurzdarm-Syndrom, die Mikrovill-Inclusion-Krankheit und durch Cholera verursachten Durchfall.
Jaguar Health (NASDAQ:JAGX) قد قدمت طلباً للحصول علىDesignation دواء يتيماً (ODD) لدى FDA لدواء Crofelemer، يستهدف علاج الإسهال لدى مرضى سرطان الثدي المصابات باستدارات دماغية ويتلقّين علاجاً مستهدفاً مع أو بدون العلاج الكيميائي القياسي.
يعتمد الطلب على اعتراف FDA بأن الاستدارات الدماغية تُعد حالة مميزة عن الأورام الأساسية، كما يتضح من سبع ODDs مُنحت بين 2015 و2024. إذا مُنحت، ستوفر ODD سبع سنوات من حصرية السوق، وخصومات ضريبية للاختبارات السريرية، وتخفيفاً من رسوم التقديم.
تخطط الشركة للسعي للحصول على تصنيفات مثل Breakthrough Therapy و/أو Fast Track لتسريع المسارات التنظيمية. Crofelemer يمتلك بالفعل ODDs لاضطرابات مثل متلازمة الأمعاء القصيرة، مرض الإدراج الميكروفيلي، والإسهال المرتبط بالكوليرا.
Jaguar Health (NASDAQ:JAGX) 已向 FDA 提交了 crofelemer 的孤儿药指示（ODD）申请，针对接受靶向治疗（有或没有标准化疗）的乳腺癌脑转移患者的腹泻治疗。
该申请利用 FDA 将脑转移视为与原发性肿瘤不同的病症的认识，2015-2024 年间已有 7 项 ODD 授予记录可证此点。若获批，ODD 将提供 七年市场独占期、临床试验税收抵免以及申报费减免。
公司计划通过争取突破性疗法（Breakthrough Therapy）和/或 Fast Track 指定来加速监管路径。Crofelemer 已持有针对短肠综合征、微绒毛包含病以及霍乱相关腹泻的 ODD。
- FDA recognition of brain metastasis as distinct condition supports potential ODD approval
- Previous successful ODDs for crofelemer in other indications demonstrate regulatory experience
- Potential for expedited regulatory pathway through Breakthrough/Fast Track designation
- Seven-year marketing exclusivity if ODD is granted
- No guarantee of ODD approval from FDA
- Clinical development and regulatory process still required even if ODD is granted
Insights
Jaguar Health's orphan drug application for crofelemer could unlock regulatory incentives and exclusivity for their cancer therapy-related diarrhea treatment.
Jaguar Health has submitted an application for orphan drug designation (ODD) for their drug crofelemer to treat diarrhea in breast cancer patients with brain metastases receiving targeted therapy. This is a strategic regulatory maneuver with multiple potential benefits.
The FDA has previously recognized brain metastasis as a distinct condition from the primary cancer site, granting seven orphan designations for such therapies between 2015-2024. This precedent strengthens Jaguar's application and indicates a viable regulatory pathway.
If granted, this ODD would provide Jaguar with significant incentives, including tax credits for clinical testing, relief from filing fees, and most importantly, seven years of marketing exclusivity. This exclusivity period creates a competitive moat that could substantially enhance the commercial value of crofelemer in this indication.
The company is also positioning to pursue expedited regulatory pathways through Breakthrough Therapy or Fast Track designations, which could accelerate approval timelines. This is particularly relevant for cancer supportive care where unmet needs are high.
Notably, crofelemer already holds orphan designations for several other conditions, demonstrating the company's expertise in navigating these regulatory channels. This submission represents a continuation of Jaguar's strategy to expand crofelemer's indications while maximizing regulatory advantages.
Potential treatment for patients with breast cancer with metastasis to the brain with crofelemer would augment the significant responder analysis results for crofelemer in the prespecified subgroup of patients with breast cancer from the OnTarget study that were reported at the Multinational Association of Supportive Care in Cancer 2025 Annual Meeting and the 2024 San Antonio Breast Cancer Symposium
SAN FRANCISCO, CA / ACCESS Newswire / September 24, 2025 / Jaguar Health (NASDAQ:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) has submitted an orphan drug designation (ODD) application to the U.S. Food and Drug Administration (FDA) for crofelemer, the company's novel prescription drug, for the treatment of diarrhea in adult patients with breast cancer that has metastasized to the brain receiving targeted therapy with or without standard chemotherapy.
Within the last ten years, the FDA's Office of Orphan Products Development (OOPD) has publicly acknowledged that brain metastasis of any cancer is considered a disease or condition separate and distinct from the primary site of origin. Accordingly, between 2015 and 2024 the OOPD awarded seven orphan drug designations for various therapies for the treatment of breast cancer that has metastasized to the brain.
"The FDA has identified the condition of breast cancer that has metastasized to the brain as an orphan indication. We have submitted this ODD application for crofelemer to treat diarrhea in these patients," said Lisa Conte, Jaguar's founder, president, and CEO. "Diarrhea is a common side effect of numerous targeted cancer therapies that can lead to dose changes, treatment delays, or cessation of treatment altogether, all of which impact patient outcomes. Given crofelemer's novel and physiological mechanism of action, we would plan to seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory pathways in the U.S. for crofelemer for this indication."
In both the US and European Union, crofelemer has previously received orphan drug designation for short bowel syndrome with intestinal failure and microvillus inclusion disease for a liquid formulation of crofelemer. Crofelemer has also been granted an orphan drug designation for treatment of diarrhea in cholera in the U.S. Orphan drug designation qualifies the drug sponsor for various development and/or regulatory incentives, such as tax credits for qualified clinical testing and relief from filing fees. Futhermore, orphan drug designation in the U.S. provides a seven-year period of marketing exclusivity to the first sponsor.
About Crofelemer
Crofelemer is a botanical (plant-based) drug extracted and purified from the red bark sap, also referred to as "dragon's blood," of the medicinal Croton lechleri tree in the Amazon Rainforest. Jaguar family company Napo Pharmaceuticals has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities.
About the Jaguar Health Family of Companies
Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan diseases.
For more information about:
Jaguar Health, visit https://jaguar.health
Napo Pharmaceuticals, visit www.napopharma.com
Napo Therapeutics, visit napotherapeutics.com
Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram
Forward-Looking Statements
Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that Napo may seek Breakthrough Therapy designation and/or Fast Track designation from the FDA to support potentially expedited regulatory pathways in the U.S. for crofelemer for the treatment of diarrhea in adult patients with breast cancer that has metastasized to the brain. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
CONTACT:
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SOURCE: Jaguar Health, Inc.
View the original press release on ACCESS Newswire
FAQ
What is Jaguar Health's (JAGX) new orphan drug designation application for?
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What other orphan drug designations does JAGX's crofelemer already have?
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