Jaguar Health Family Company Napo Pharmaceuticals to Meet with FDA to Discuss Potential Regulatory Pathways for Crofelemer for Treatment of Ultrarare Pediatric Indication Microvillus Inclusion Disease (MVID)
Jaguar Health (NASDAQ:JAGX) subsidiary Napo Pharmaceuticals announced plans to meet with the FDA regarding crofelemer's development for treating Microvillus Inclusion Disease (MVID), an ultrarare pediatric disorder. Initial proof-of-concept results from an ongoing investigator-initiated trial in Abu Dhabi demonstrated that crofelemer reduced required total parenteral nutrition by up to 27% in the first MVID patient.
The company aims to discuss potential expedited regulatory pathways for this rare orphan indication. An abstract describing the trial results has been accepted for presentation at the NASPGHAN 2025 Annual Meeting in Chicago. Napo is currently conducting a placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients across the U.S., EU, and Middle East/North Africa regions.
Jaguar Health (NASDAQ:JAGX), tramite la sua controllata Napo Pharmaceuticals, ha annunciato l'intenzione di incontrare la FDA per discutere lo sviluppo di crofelemer nel trattamento della Microvillus Inclusion Disease (MVID), una rara patologia pediatrica. I risultati preliminari di un trial iniziato da un investigatore ad Abu Dhabi mostrano che crofelemer ha ridotto la necessità di nutrizione parenterale totale fino al 27% nel primo paziente con MVID.
L'azienda intende esplorare possibili percorsi regolatori accelerati per questa indicazione orfana. Un abstract con i risultati è stato accettato per la presentazione al NASPGHAN 2025 Annual Meeting di Chicago. Napo sta inoltre conducendo uno studio di Fase 2, controllato con placebo, su pazienti pediatrici con MVID in USA, UE e regioni Medio Oriente/Nord Africa.
Jaguar Health (NASDAQ:JAGX), a través de su filial Napo Pharmaceuticals, anunció planes para reunirse con la FDA para tratar el desarrollo de crofelemer en el tratamiento de la Microvillus Inclusion Disease (MVID), un trastorno pediátrico ultrarraro. Resultados iniciales de prueba de concepto de un ensayo iniciado por un investigador en Abu Dhabi mostraron que crofelemer redujo la necesidad de nutrición parenteral total hasta en 27% en el primer paciente con MVID.
La compañía pretende discutir posibles vías regulatorias aceleradas para esta indicación huérfana. Un resumen que describe los resultados del ensayo fue aceptado para su presentación en la NASPGHAN 2025 Annual Meeting en Chicago. Napo está llevando a cabo un estudio de Fase 2 controlado con placebo de crofelemer en pacientes pediátricos con MVID en EE. UU., la UE y las regiones de Oriente Medio/Norte de África.
Jaguar Health (NASDAQ:JAGX)의 자회사 Napo Pharmaceuticals는 초희귀 소아 질환인 미세융모 포함체 질환(MVID) 치료를 위한 크로펠머(crofelemer) 개발 관련 FDA와의 회의를 계획한다고 발표했습니다. 아부다비에서 진행 중인 연구자 주도 시험의 초기 개념 증명 결과에 따르면, 크로펠머는 첫 MVID 환자에서 총 비경구영양(TPN) 필요량을 최대 27%까지 줄였습니다.
회사는 이 희귀 질환에 대해 신속 심사 등 잠재적 규제 가속 경로를 논의할 계획입니다. 해당 시험 결과를 설명한 초록이 시카고에서 열리는 NASPGHAN 2025 Annual Meeting에 발표로 채택되었습니다. Napo는 현재 미국, EU 및 중동/북아프리카 지역의 소아 MVID 환자를 대상으로 위약 대조 제2상 시험을 진행 중입니다.
La filiale Napo Pharmaceuticals de Jaguar Health (NASDAQ:JAGX) a annoncé son intention de rencontrer la FDA pour discuter du développement du crofelemer dans le traitement de la Microvillus Inclusion Disease (MVID), une maladie pédiatrique ultra-rare. Les premiers résultats de preuve de concept d'un essai initié par un investigateur à Abou Dhabi montrent que le crofelemer a réduit les besoins en nutrition parentérale totale jusqu'à 27% chez le premier patient atteint de MVID.
La société souhaite examiner d'éventuelles voies réglementaires accélérées pour cette indication orpheline. Un résumé décrivant les résultats de l'essai a été accepté pour présentation lors du NASPGHAN 2025 Annual Meeting à Chicago. Napo conduit actuellement un essai de Phase 2 contrôlé par placebo du crofelemer chez des enfants atteints de MVID aux États-Unis, dans l'UE et au Moyen-Orient/Nord de l'Afrique.
Die Tochtergesellschaft Napo Pharmaceuticals von Jaguar Health (NASDAQ:JAGX) gab Pläne bekannt, sich mit der FDA zu treffen, um die Entwicklung von Crofelemer zur Behandlung der Microvillus Inclusion Disease (MVID), einer ultrararen pädiatrischen Erkrankung, zu besprechen. Erste Proof-of-Concept-Ergebnisse aus einer laufenden, vom Prüfärzteteam initiierten Studie in Abu Dhabi zeigten, dass Crofelemer beim ersten MVID-Patienten den Bedarf an totaler parenteraler Ernährung um bis zu 27% verringerte.
Das Unternehmen beabsichtigt, mögliche beschleunigte regulatorische Wege für diese Orphan-Indikation zu erörtern. Ein Abstract mit den Studienergebnissen wurde für eine Präsentation auf der NASPGHAN 2025 Annual Meeting in Chicago angenommen. Napo führt derzeit eine placebokontrollierte Phase-2-Studie mit Crofelemer bei pädiatrischen MVID-Patienten in den USA, der EU sowie in der Region Naher Osten/Nordafrika durch.
- Initial trial results showed up to 27% reduction in required total parenteral nutrition for MVID patients
- Abstract accepted for presentation at major industry conference (NASPGHAN 2025)
- Potential for expedited regulatory approval pathway due to ultrarare disease status
- Ongoing Phase 2 study across multiple global regions
- Only one patient data point available so far
- No currently approved drug treatments for MVID, indicating regulatory uncertainty
Insights
Napo's meeting with FDA about crofelemer for MVID represents meaningful progress for an untreatable ultrarare pediatric disease.
Napo Pharmaceuticals' upcoming FDA meeting represents a significant milestone in their development of crofelemer for Microvillus Inclusion Disease (MVID), an ultrarare pediatric disorder with no approved treatments. The company will specifically discuss potential expedited regulatory pathways based on promising early clinical data.
The most compelling aspect of this development is the 27% reduction in total parenteral nutrition (TPN) requirements observed in the first MVID patient treated in their Abu Dhabi investigator-initiated trial. This is particularly meaningful because MVID patients are completely dependent on TPN for survival, which carries significant complications including liver damage, line infections, and poor quality of life.
For an ultrarare disease like MVID, even small patient populations showing clinical benefit can potentially support regulatory approval pathways. The company is strategically pursuing this opportunity by:
- Conducting their proof-of-concept study in Abu Dhabi
- Running a placebo-controlled Phase 2 study across multiple regions (US, EU, Middle East/North Africa)
- Seeking FDA guidance on expedited pathways given the disease rarity
The presentation of their initial results at the upcoming NASPGHAN meeting will provide important visibility among pediatric gastroenterologists who treat these patients. If crofelemer continues to demonstrate TPN reduction benefits, it could represent the first therapeutic option for this devastating condition where management currently relies exclusively on parenteral support.
As announced, initial proof-of-concept results from the ongoing investigator-initiated trial in Abu Dhabi show crofelemer reduced the required total parenteral nutrition in the first participating MVID patient by up to
SAN FRANCISCO, CA / ACCESS Newswire / August 19, 2025 / Jaguar Health, Inc.(NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today announced that it plans to meet with the U.S. Food and Drug Administration (FDA) to discuss the company's ongoing clinical development program for crofelemerfor the treatment of microvillus inclusion disease (MVID), an ultrarare pediatric disorder. Members of Napo's Scientific Advisory Board will join Napo and Jaguar representatives at the meeting.
"We're very pleased that Napo has been granted a meeting with the FDA to discuss Napo's development plans for crofelemer for MVID - a devastating pediatric disease characterized by severe malabsorption that requires life-sustaining parenteral support to meet the nutritional, fluid and electrolyte requirements of the child, and for which there no approved drug treatments," said Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "A core Napo goal for this meeting is to obtain input from the FDA on the clinical program and potential expedited regulatory pathways for this rare orphan indication."
As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, the initial proof-of-concept results of the ongoing investigator-initiated trial (IIT) of a novel crofelemer powder formulation for oral solution in Abu Dhabi in the United Arab Emirates show that crofelemer reduced the required total parenteral nutrition (TPN) and supplementary intravenous fluids in the first participating MVID patient by up to
Jaguar, through Napo, is supporting the independent proof-of-concept IIT in pediatric intestinal failure (IF) patients at Sheikh Khalifa Medical City in Abu Dhabi, and is conducting the placebo-controlled Phase 2 study of crofelemer in pediatric MVID patients with IF at sites in the U.S., European Union, and Middle East/North Africa regions under appropriate regulatory approvals in each of these geographies.
"Given the ultrarare nature of MVID, and the groundbreaking initial proof-of-concept results from the IIT in Abu Dhabi, even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval," said Lisa Conte, Jaguar's Founder and CEO.
Based on the initial findings from the IIT in Abu Dhabi, crofelemer's paradigm-shifting mechanism of action has the potential to provide a novel therapeutic option to reduce parenteral support and associated complications in MVID patients.
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. Jaguar family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics S.p.A. focus on the development and commercialization of novel crofelemer powder for oral solution for the treatment of rare and orphan gastrointestinal disorders with intestinal failure, including MVID and short bowel syndrome.
For more information about:
Jaguar Health, visit https://jaguar.health
Napo Pharmaceuticals, visit www.napopharma.com
Napo Therapeutics, visit napotherapeutics.com
Forward-Looking Statements
Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that Napo personnel will meet with the FDA to discuss Napo's development plans for crofelemer for MVID, statements regarding Jaguar's expectation that an abstract describing the results of the investigator-initiated trial in Abu Dhabi will be presented at NASPGHAN 2025, Jaguar's expectation that even a small number of MVID patients showing benefit with crofelemer may allow Napo to explore pathways for expedited regulatory approval, and Jaguar's expectation that crofelemer's paradigm-shifting mechanism of action has the potential to provide a novel therapeutic option to reduce parenteral support and associated complications in MVID patients. 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 several 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.
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