Jaguar Health Reports Promising Results for Crofelemer in Rare Pediatric Intestinal Failure Conditions
Key Insights
Jaguar Health's Napo Pharmaceuticals (search) announced acceptance of two late-breaking abstracts at ESPGHAN demonstrating safety and effectiveness of liquid oral crofelemer for pediatric intestinal failure (search) patients.
Crofelemer showed reductions in parenteral support needs for patients with microvillus inclusion disease (search) (MVID) and short bowel syndrome (search), with one infant treated for over 6 months and other patients for over 1 year.
The company has completed enrollment for a randomized clinical trial in pediatric MVID patients with plans for NDA filing in mid-2027, targeting a rare disease affecting approximately 200 patients globally.
Jaguar Health's subsidiary Napo Pharmaceuticals (search) announced the acceptance of two late-breaking abstracts for presentation at the 58th Annual Meeting of ESPGHAN, showcasing promising results for liquid oral crofelemer in treating pediatric intestinal failure (search) (IF) caused by microvillus inclusion disease (search) (MVID) and short bowel syndrome (search) (SBS). The data represents significant progress for patients with these ultrarare conditions who currently depend on life-sustaining parenteral support.
Clinical Trial Results Show Reduced Parenteral Support Needs
The abstracts detail outcomes from two separate investigator-initiated trials involving pediatric IF patients receiving crofelemer as adjunctive therapy. One infant MVID patient has been treated with crofelemer alongside parenteral support for more than six months, while other pediatric patients with IF due to MVID and/or SBS received crofelemer adjunctive therapy for over one year with reduced amounts of parenteral support.
According to Dr. Pravin Chaturvedi, Napo's Chief Scientific Officer and Chair of the company's Scientific Advisory Board, "Crofelemer has demonstrated reductions in parenteral support needs for IF patients with both MVID and SBS. Crofelemer has been well tolerated, and the pediatric IF patients are gaining weight and height, demonstrating reductions in their needs for PS, especially when adjusted for body weight."
Addressing Critical Unmet Medical Need
MVID represents a congenital enteropathy with a lethal natural history if not treated with life-sustaining and lifelong total parenteral nutrition (TPN) with or without supplemental intravenous fluids. The condition is estimated to have a prevalence of approximately 200 patients globally. According to the company, crofelemer is currently being used to treat approximately 4% of the prevalent MVID patients, including additional patients enrolled in an ongoing blinded study.
The reduction in parenteral support needs carries significant clinical implications, as TPN is associated with serious toxicities including life-threatening infections, metabolic complications, liver and kidney function problems, reduction in normal growth parameters, and cognitive functioning impacts. Many intestinal failure (search) patients require parenteral support up to seven days a week, sometimes for 20 hours or more per day.
Regulatory Progress and Development Pipeline
Jaguar has received orphan drug designations for both MVID and SBS in the United States and European Union. The company has completed enrollment of pediatric MVID patients in an ongoing randomized clinical trial with plans for a New Drug Application (NDA) filing in mid-2027.
Lisa Conte, the company's founder and CEO, emphasized the drug's safety profile: "Crofelemer continues to have an impressive safety profile while demonstrating disease progression modification for the fragile pediatric IF patient population. The company has received interest and support from the parents of these IF patients, which has been heartening and inspirational."
Expanding Clinical Development
Beyond the pediatric studies, Jaguar's family company Napo Therapeutics (search) S.P.A. is continuing to enroll adult patients with IF due to SBS using the same formulation of crofelemer. This randomized double-blind placebo-controlled trial is being conducted at multiple sites in Germany and Italy. An investigational study in adult SBS-IF patients is also being partially supported by the company in the United States.
The clinical development program represents a comprehensive approach to addressing intestinal failure (search) across different patient populations and underlying conditions, with crofelemer positioned as a potential adjunctive therapy to reduce dependence on parenteral support while maintaining patient safety and promoting normal growth parameters.