All Randomized Pediatric Patients to Continue in the Single-Blind Extension Phase of Jaguar Health's Pivotal Clinical Trial of Crofelemer for Microvillus Inclusion Disease (MVID) with Intestinal Failure (IF)
All randomized pediatric MVID patients move into an extension phase of crofelemer treatment, supporting Jaguar’s planned mid‑2027 NDA filing.
Rhea-AI Summary
Jaguar Health (JAGX) reported that all randomized pediatric patients with microvillus inclusion disease (MVID) in Napo Pharmaceuticals' pivotal crossover trial have entered the single-blind extension phase to receive blinded doses of crofelemer as adjunctive therapy to parenteral support (PS).
The ongoing trial is being conducted at multiple centers in the United States, European Union, and United Arab Emirates and is expected to support a New Drug Application (NDA) filing for the MVID indication in mid-2027. An independent Data Monitoring Committee evaluated safety and tolerability during the randomized double-blind crossover period and authorized continuation of active crofelemer treatment in the extension phase. Previous investigational data in pediatric intestinal failure (IF) due to MVID and short bowel syndrome with IF (SBS-IF) showed substantial reductions in weekly PS needs, including up to a 48% reduction in PS normalized to body weight in one MVID patient and up to 40% in two SBS-IF patients after more than 12 months of high-dose oral crofelemer.
Positive
- All randomized MVID patients entered the single-blind extension phase with blinded crofelemer dosing
- Prior long-term crofelemer use cut PS needs by up to 48% in one pediatric MVID patient
- Prior long-term crofelemer use cut PS needs by up to 40% in two pediatric SBS-IF patients
- Independent Data Monitoring Committee cleared continued active crofelemer treatment after safety review
- IF program expected to deliver mid-2027 NDA filing for MVID and completion of a Phase 2 SBS-IF study
Negative
- None.
Key Figures
- Planned NDA filing
- mid-2027
- MVID indication
- PS reduction
- up to 48%
- One MVID patient after >12 months of liquid oral high-dose crofelemer therapy
- PS reduction
- up to 40%
- Two pediatric SBS-IF patients after >12 months of liquid oral high-dose crofelemer therapy
- Treatment duration
- >12 months
- Liquid oral high-dose crofelemer investigations
- Worldwide prevalence
- 200 patients
- Estimated worldwide MVID prevalence
Previous Clinical trial Reports
-
First pediatric patient entered the single-blind extension phase supporting the planned mid-2027 NDA filing
-
FDA feedback supported a single-blind extension assessing longer-term clinical benefit
-
Amended protocol was submitted to FDA for a possible expedited crofelemer approval pathway
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Key Terms
single-blind extension phase medical
parenteral support medical
total parenteral nutrition medical
new drug application regulatory
breakthrough therapy designation regulatory
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Continuation of treatment with blinded doses of crofelemer in the extension phase of the trial will support company's plans to file an NDA in mid-2027 for MVID, an ultrarare pediatric disease
SAN FRANCISCO, CA / ACCESS Newswire / September 10, 2026 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today announced that all randomized pediatric MVID patients in Napo's pivotal crossover clinical trial have entered the single-blind extension phase to receive blinded doses of crofelemer to support longer-term safety and efficacy for crofelemer powder for oral solution as adjunctive therapy to parenteral support (PS). The trial is ongoing at multiple centers in the United States, the European Union, and the United Arab Emirates. This trial is expected to support a New Drug Application (NDA) filing for the treatment of the MVID indication in mid-2027.
Patients with intestinal failure (IF), including those with MVID or short bowel syndrome (SBS), cannot orally absorb adequate nutrients, electrolytes and fluids, and require lifelong total parenteral nutrition (TPN) with or without supplemental intravenous fluids, which together comprise PS, for up to seven days per week and up to 20+ hours per day. While PS is supportive, palliative and lifesaving, it is associated with serious, sometimes fatal complications.
Napo remains sharply focused on its ongoing global development program for oral crofelemer as adjunctive therapy to PS for pediatric rare IF diseases, including MVID and short bowel syndrome with IF (SBS-IF). In June 2026, as announced, data was presented at the 58th Annual European Society for Pediatric Gastroenterology, Hepatology & Nutrition (ESPGHAN) Meeting in Lille, France from ongoing investigational studies with oral liquid crofelemer in pediatric patients with IF due to MVID and SBS-IF. The results of ongoing investigations of liquid oral crofelemer formulations demonstrate substantial reductions in weekly PS needs as well as in PS normalized to body weight in pediatric IF patients dosed orally for more than one year, with no significant clinical or laboratory abnormalities. In one MVID patient, the weekly PS requirements normalized to body weight have been reduced by up to
"We are pleased that all randomized pediatric MVID patients will participate in the single-blind extension phase with blinded doses of crofelemer powder for oral solution. An independent Data Monitoring Committee evaluated the safety and tolerability of crofelemer during the randomized double-blind crossover period of the trial and authorized continuation of active crofelemer treatment for a longer period in the extension phase," said Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "As previously reported at ESPGHAN 2026, long-term crofelemer treatment has been well tolerated and demonstrated significant reductions in PS normalized to body weight in two open-label investigations in pediatric MVID patients."
"Our IF program is expected to continue to provide clinical milestones, including an NDA filing for MVID and the completion of a Phase 2 study in SBS-IF by mid-2027. The totality of our IF program with the same powder for liquid crofelemer formulation is the subject of business development discussions with the potential to bring in non-dilutive funds from potential licensee partners," said Lisa Conte, Jaguar's founder, president, and CEO. "Upon completion of the trial, data from this study may serve as the basis for a Breakthrough Therapy Designation application. Because MVID is a devastating ultrarare pediatric disorder with a lethal natural history - and an estimated worldwide prevalence of only 200 patients - a trial of crofelemer in just a small number of MVID patients is expected to be clinically meaningful and significant."
About Crofelemer
Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. 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. Jaguar family companies Napo Pharmaceuticals, Inc. (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 microvillus inclusion disease and short bowel syndrome.
For more information about:
Jaguar Health, visit https://jaguar.health
Napo Pharmaceuticals, visit 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 its intestinal failure program will continue to provide clinical milestones, including an NDA filing for MVID and the completion of a Phase 2 study in SBS-IF by mid-2027, Jaguar's expectation that the opportunity may exist to bring in non-dilutive funds from potential licensee partners to support the intestinal failure program, Jaguar's plans to pursue Breakthrough Therapy Designation for crofelemer for the indication of MVID, and Jaguar's expectation that a trial of crofelemer in a small number of MVID patients may be clinically meaningful and significant. 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:
hello@jaguar.health
Jaguar-JAGX
SOURCE: Jaguar Health, Inc.
View the original press release on ACCESS Newswire
FAQ
What is the purpose of the single-blind extension phase for crofelemer in MVID?
The single-blind extension phase allows all randomized pediatric MVID patients to continue receiving blinded doses of crofelemer powder for oral solution as adjunctive therapy to parenteral support. This phase is intended to support the collection of longer-term safety and efficacy data for crofelemer in this ultrarare intestinal failure indication.
How does crofelemer treatment relate to Jaguar Health’s regulatory plans?
The ongoing pivotal trial, including the single-blind extension phase, is expected to support a New Drug Application filing for crofelemer for the MVID indication in mid-2027. The company also expects completion of a Phase 2 study in short bowel syndrome with intestinal failure (SBS-IF) by mid-2027, using the same crofelemer powder for liquid formulation.
What clinical benefits have been observed with liquid oral crofelemer in pediatric intestinal failure?
In ongoing investigational studies, long-term dosing with liquid oral high-dose crofelemer in pediatric intestinal failure patients has shown substantial reductions in weekly parenteral support (PS) needs, including reductions in PS normalized to body weight. Reported examples include up to a 48% reduction in one MVID patient and up to a 40% reduction in two SBS-IF patients after more than 12 months of therapy, with no significant clinical or laboratory abnormalities observed.
Could data from this MVID trial support any additional regulatory designations?
The company said that, upon completion of the trial, data from the study may serve as the basis for an application for Breakthrough Therapy Designation for crofelemer in MVID.
Where is the pivotal crofelemer trial for MVID being conducted?
The pivotal crossover clinical trial for crofelemer in pediatric MVID patients with intestinal failure is ongoing at multiple centers in the United States, the European Union, and the United Arab Emirates.
What business development opportunities are associated with Jaguar’s intestinal failure program?
The company stated that the totality of its intestinal failure program, based on the same crofelemer powder for liquid formulation, is the subject of business development discussions with potential licensee partners, with the potential to bring in non-dilutive funds.
What are MVID and SBS-IF and why do patients need parenteral support?
Patients with intestinal failure, including those with microvillus inclusion disease (MVID) or short bowel syndrome (SBS), cannot orally absorb adequate nutrients, electrolytes, and fluids. They require lifelong total parenteral nutrition with or without supplemental intravenous fluids, collectively referred to as parenteral support, which can be needed up to seven days per week and for more than 20 hours per day.