Jaguar Health Inc. (NASDAQ: JAGX) announced it held preliminary discussions with the FDA's Division of Gastroenterology to evaluate breakthrough therapy designation for oral liquid crofelemer as treatment for pediatric patients with intestinal failure due to microvillus inclusion disease (MVID).

MVID is described as an ultrarare congenital disorder with a lethal natural history that requires lifelong parenteral support. The company stated there are currently no available therapies for the condition other than lifelong parenteral support.

According to the announcement, crofelemer demonstrated preliminary clinical proof of concept in two open-label studies involving pediatric MVID patients, showing meaningful reductions in weekly parenteral support requirements. Initial results from these studies have been accepted for presentation at the 58th Annual ESPGHAN Meeting in June 2026.

The company reported that crofelemer is currently being used to treat approximately 4% of the estimated living pediatric MVID patients. A pivotal randomized double-blind placebo-controlled clinical study in pediatric MVID subjects has completed enrollment.

"Crofelemer has demonstrated meaningful reductions in PS needs for pediatric IF patients with MVID," said Dr. Pravin Chaturvedi, Napo's Chief Scientific Officer. "Crofelemer has been well tolerated, and the pediatric MVID patients are gaining weight and height and demonstrating reductions in their daily and weekly needs for PS."

Jaguar and its family company Napo Pharmaceuticals have received orphan drug designations in the United States and European Union for crofelemer for both MVID and short bowel syndrome with intestinal failure.

Breakthrough therapy designation is an FDA program designed to expedite development and review of drugs treating serious or life-threatening conditions where preliminary clinical evidence indicates substantial improvement over available therapy.