Jaguar Health's crofelemer shows promise in reducing parenteral support for intestinal failure patients

SAN FRANCISCO, Jaguar Health, Inc., a pharmaceutical company focused on developing plant-based prescription medicines, announced that its subsidiary Napo Pharmaceuticals is providing its novel crofelemer powder for oral solution to an infant patient suffering from congenital sodium diarrhea (CSD), a rare intestinal failure (IF) disease caused by a GUCY2C gene mutation. This compassionate use case marks an important step in the company's ongoing global development program for crofelemer as an adjunctive therapy to parenteral support (PS) for pediatric and adult IF diseases, including microvillus inclusion disease (MVID) and short bowel syndrome with IF (SBS-IF).

Crofelemer, a novel, plant-based prescription medicine, has shown promising results in reducing the need for parenteral support in pediatric patients with MVID and SBS-IF. Data from ongoing investigational studies presented at the 58th Annual European Society for Pediatric Gastroenterology, Hepatology & Nutrition (ESPGHAN) Meeting in Lille, France, demonstrated that liquid oral crofelemer formulations significantly reduced weekly PS needs and PS normalized to body weight in pediatric patients. In one MVID patient, weekly PS requirements normalized to body weight decreased by up to 48% after more than 12 months of liquid oral high-dose crofelemer therapy. Similar reductions were observed in two SBS-IF patients, with weekly PS requirements normalized to body weight reduced by up to 40%.

Intestinal failure patients, including those with MVID or short bowel syndrome, cannot absorb nutrients, electrolytes, and fluids orally, necessitating lifelong total parenteral nutrition (TPN) with or without supplemental intravenous fluids. While parenteral support is essential, it is associated with serious complications. Patients with a GUCY2C mutation and CSD face significant challenges, particularly in pediatric patients where growth and developmental status are heavily impacted. The infant receiving crofelemer is currently under parenteral support and is using crofelemer as an adjunctive therapy to reduce daily and weekly PS needs.

"Supporting physicians who need an effective adjunctive therapy to manage the unmet medical needs of their specific pediatric IF patients is a priority for us," stated Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "Our liquid oral crofelemer formulation has been well-tolerated with no significant clinical or laboratory abnormalities in selected pediatric IF patients."

Jaguar Health remains committed to advancing its global IF development program, with plans to file a New Drug Application (NDA) for crofelemer for MVID in mid-2027. The company has completed the randomized double-blind treatment phase of its pivotal clinical trial for pediatric MVID patients, and the resulting data may support a Breakthrough Therapy Designation application. Given the ultrarare nature of MVID, with an estimated worldwide prevalence of only 200 patients, a trial involving a small number of patients is expected to be clinically meaningful.

Crofelemer, derived from the red bark sap of the Croton lechleri tree in the Amazon Rainforest, is produced through a sustainable harvesting program under fair trade practices to ensure high quality, ecological integrity, and support for indigenous communities. The company's development efforts also include its subsidiary Napo Therapeutics, which focuses on the commercialization of crofelemer for the treatment of rare gastrointestinal disorders with IF.