FDA-Authorized Expanded Access Programs Utilizing Jaguar Health's Novel Crofelemer Powder for Oral Solution to Treat Two Pediatric Intestinal Failure Patients with Microvillus Inclusion Disease
Rhea-AI Summary
Jaguar Health (NASDAQ:JAGX) announced FDA authorization for expanded access programs using their novel crofelemer powder for oral solution to treat two pediatric intestinal failure patients with microvillus inclusion disease (MVID). The programs operate under separate Single-Patient Investigational New Drug applications in the US.
Initial proof-of-concept results from ongoing investigator-initiated trials showed that crofelemer reduced required total parenteral nutrition (TPN) by up to 27% in MVID patients and up to 12.5% in SBS-IF patients. The company expects additional trial results in 2025 and data from placebo-controlled studies in 2026.
Jaguar aims to pursue expedited regulatory pathways and potential early access programs in Europe, targeting a market of approximately 200 MVID patients worldwide and 10,000-20,000 SBS patients in both the US and Europe.
Positive
- Initial trial results showed significant TPN reduction of up to 27% in MVID patients
- Potential for expedited regulatory pathways in US and EU markets
- Opportunity for early reimbursed patient access in EU countries by 2026
- Addresses unmet medical needs with no current approved treatments for MVID
Negative
- Small patient population with only about 200 MVID patients worldwide
- Final clinical trial data not expected until 2026
- Early access programs not available in the US market
Insights
Jaguar's crofelemer shows promising early results for rare intestinal diseases, advancing toward potential regulatory pathways and early access programs.
Jaguar Health's announcement reveals strategic advancement in their rare disease program through FDA-authorized expanded access for their novel crofelemer powder formulation to treat two pediatric patients with microvillus inclusion disease (MVID). This compassionate use approval signifies regulatory acknowledgment of the drug's potential benefit in an ultrarare condition affecting approximately 200 patients worldwide with no currently approved treatments.
The preliminary efficacy signals from their ongoing investigator-initiated trials are particularly noteworthy, with data showing crofelemer reduced total parenteral nutrition (TPN) requirements by up to 27% in an MVID patient and 12.5% in a short bowel syndrome with intestinal failure (SBS-IF) patient. These reductions are clinically meaningful as TPN dependence carries significant complications including infections, metabolic issues, and organ damage.
The company has strategically positioned crofelemer for potential accelerated approval pathways through multiple clinical development approaches: two independent proof-of-concept trials, two placebo-controlled studies, and these expanded access programs. This multi-pronged approach increases their chances of generating compelling evidence for regulatory submissions while providing treatment access to patients with urgent unmet needs.
Most significantly, Jaguar appears to be pursuing a dual-continent strategy, with specific emphasis on European early access programs that could enable commercialization and reimbursement as early as 2026, prior to full regulatory approval. This approach could generate revenue while the company simultaneously pursues FDA Breakthrough Therapy designation and EMA PRIME status, potentially accelerating the traditional approval timeline for these serious conditions.
Jaguar's strategic expanded access programs could accelerate revenue generation from crofelemer for rare diseases while pursuing full approval.
This expanded access authorization for Jaguar's crofelemer represents a significant development in their commercialization strategy for ultrarare and rare disease markets. The company is navigating a sophisticated regulatory pathway that could potentially lead to early revenue generation through European reimbursed early access programs by 2026 - notably before full regulatory approval.
The addressable market, while small, represents a high-value opportunity characteristic of orphan drug economics. With MVID affecting approximately 200 patients worldwide and SBS affecting 10,000-20,000 patients in both the US and Europe, Jaguar is targeting conditions with substantial unmet needs and limited competition, typically supporting premium pricing models.
Preliminary efficacy data showing TPN reduction of up to 27% in MVID patients is particularly meaningful from a healthcare economics perspective. TPN dependency is extraordinarily expensive, with annual costs potentially exceeding $100,000-$200,000 per patient when accounting for hospitalizations due to complications. Any reduction in TPN requirements could substantially reduce healthcare system costs, strengthening the reimbursement case for crofelemer.
The company's parallel pursuit of multiple regulatory pathways (FDA Breakthrough Therapy, EMA PRIME program) alongside clinical development demonstrates strategic resource allocation to accelerate time-to-market. This approach is particularly valuable for small biopharmaceutical companies where capital efficiency is crucial. The expanded access programs serve multiple strategic purposes: generating additional clinical evidence, building physician familiarity with the product, and potentially creating patient advocacy for approval - all while fulfilling their humanitarian mission of addressing these devastating conditions.
The two programs are being conducted under separate Single-Patient Investigational New Drug (sIND) applications in the United States
SAN FRANCISCO, CA / ACCESS Newswire / September 30, 2025 / Jaguar Health (NASDAQ:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) is providing the company's novel crofelemer powder for oral solution for use in two expanded access programs, authorized by the U.S. Food and Drug Administration (FDA), to treat pediatric intestinal failure patients with microvillus inclusion disease (MVID).
"Napo is committed to providing the novel crofelemer formulation as an investigational drug as deemed medically necessary by the physician caregiver for these two patients, intended for mitigating the sequela from MVID disease progression," said Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "We wish both pediatric patients the best outcome and we are grateful to have this collaboration with the patients' physician."
Jaguar, through Jaguar family companies Napo and Napo Therapeutics, is currently supporting two independent proof-of-concept investigator-initiated trials (IIT), and conducting two placebo-controlled clinical studies, of crofelemer in patients with intestinal failure due to the ultrarare disease MVID and the rare disease indication short bowel syndrome with intestinal failure (SBS-IF) in the United States, European Union, and/or Middle East/North Africa regions under appropriate regulatory approvals in each of these geographies.
As announced, and as presented April 26, 2025 at the Annual ELITE PED-GI Congress, the initial proof-of-concept results of the ongoing 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
Additional proof-of-concept results from IITs of crofelemer for MVID and SBS-IF are expected later in 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs. Data from both above-referenced placebo-controlled clinical studies is expected in 2026.
In accordance with the guidelines of specific European Union countries, published data from clinical investigations could support reimbursed early patient access to crofelemer for these debilitating conditions in 2026 while the company pursues approval of crofelemer for MVID and SBS-IF from the European Medicines Agency (EMA) and the FDA. Participation in early access programs, which do not exist in the U.S., provides an opportunity for reimbursement while impacting the morbidity and high cost of care for these chronic unmet needs. Additionally, the company expects that if even just a very small number of MVID patients show benefit with crofelemer, this may potentially allow expedited regulatory pathways in the U.S. and other regions, including qualifying crofelemer for participation in PRIME, an EMA program providing enhanced interaction and early dialogue with drug developers of novel medicines targeting unmet medical needs, and in the FDA's Breakthrough Therapies program.
Patients with MVID and SBS-IF suffer from devastating diarrhea and dehydration caused by these debilitating, lifelong conditions. These patients are frequently on TPN for as long as 20 hours a day, seven days a week - and TPN carries a significant risk of morbidity, infections, metabolic complications, liver and kidney problems, and neurodevelopmental delay. There are no approved drug treatments for MVID, an ultrarare pediatric disease with an estimated prevalence of about 200 patients worldwide. Short bowel syndrome (SBS) affects approximately 10,000 to 20,000 people in the U.S., according to the Crohn's & Colitis Foundation, and it is estimated that the population of SBS patients in Europe is approximately the same size.
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
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Forward-Looking Statements
Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that additional proof-of-concept results from IITs of crofelemer for MVID and SBS-IF will be available later in 2025 and will provide additional preliminary data on the safety and potential effectiveness of crofelemer for these highly unmet clinical needs, Jaguar's expectation that data from both above-referenced placebo-controlled clinical studies will be available in 2026, Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations could support reimbursed early patient access to crofelemer for MVID and SBS-IF in 2026 while the company pursues approval of crofelemer for MVID and SBS-IF from the EMA and the FDA, and Jaguar's expectation that, if even just a very small number of MVID patients show benefit with crofelemer, this may potentially allow expedited regulatory pathways in the U.S. and other regions, including qualifying crofelemer for participation in the EMA's PRIME program and the FDA's Breakthrough Therapies program. 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
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