Jaguar Health's Crofelemer: New Data for Rare Disease Treatment

Jaguar Health, Inc. has announced that proof-of-concept data for its drug Crofelemer will be presented at the ELITE PED-GI Congress in Abu Dhabi in April 2025. Crofelemer, an innovative plant-based anti-secretory medication, holds Orphan Drug Designation by the FDA and EMA for treating short bowel syndrome (SBS) and microvillus inclusion disease (MVID).

Currently, no approved drug treatments exist for MVID, a severe pediatric condition necessitating significant nutritional and fluid management. Preliminary results from an ongoing study indicate Crofelemer's potential to improve nutrient absorption and reduce reliance on parenteral nutrition (TPN), thus possibly enhancing patient quality of life.

Conducted by Dr. Mohamad Miqdady, the research assesses if Crofelemer can safely be used in children with MVID, SBS-IF, and other rare disorders. Success could pave the way for regulatory approval and early patient access through the EMA's PRIME and FDA's Breakthrough Therapies programs.