Jaguar Health Inc. (NASDAQ: JAGX) announced that the first pediatric patient with Microvillus Inclusion Disease has entered the active treatment extension phase of a pivotal clinical trial for liquid oral crofelemer. The patient completed the randomized double-blind crossover period and will now participate in a single-blind extension to evaluate longer-term safety and efficacy.

The trial is conducted at multiple centers in the United States, European Union, and United Arab Emirates with regulatory approvals. MVID is a rare congenital enteropathy causing intestinal failure from early childhood, requiring patients to receive lifelong parenteral support including total parenteral nutrition.

According to the company, no approved drug treatments currently exist for MVID. The disease has a lethal natural history without life-sustaining parenteral support, and patients typically require daily support for 20 hours or more per day.

"The decision to allow pediatric MVID patients to continue into the active treatment only single-blind extension phase after completion of the randomized double-blind crossover period is made by an independent data monitoring committee," said Dr. Pravin Chaturvedi, Chief Scientific Officer of Napo Pharmaceuticals and Jaguar Health.

Jaguar's subsidiary Napo Pharmaceuticals plans to complete the clinical trial to support a New Drug Application filing in mid-2027, subject to study completion and regulatory alignment. The company will include data from two additional MVID patients being evaluated under expanded access protocols.

Jaguar has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency for crofelemer for MVID treatment. The company estimates that oral liquid crofelemer treatment is being used as adjunctive therapy in up to 4% of the global MVID patient population.