In Support of Possible Expedited Approval Pathway for Crofelemer for Treatment of Microvillus Inclusion Disease (MVID), Jaguar Health Submits Amended Protocol to FDA for Ongoing Placebo-Controlled Clinical Trial

Core Insights - Crofelemer has the potential to extend the lives of patients with microvillus inclusion disease (MVID), a rare genetic disorder affecting infants and children [1] - The company has submitted an amended protocol to the FDA for a placebo-controlled clinical trial of crofelemer in pediatric MVID patients, aiming for expedited approval [1] - MVID patients face a lethal natural history and require parenteral support, which can be reduced by up to 37% with crofelemer, marking a significant breakthrough [1] Company Developments - Jaguar Health, Inc. and its subsidiary Napo Pharmaceuticals are actively pursuing the development of crofelemer for treating intestinal failure in MVID patients [1] - The ongoing clinical trial is a critical step towards obtaining approval for a treatment that currently has no approved options in any region [1] Industry Context - The lack of approved treatments for MVID highlights a significant unmet medical need in the pediatric population affected by this disorder [1] - The potential reduction of parenteral support by crofelemer could alleviate associated toxicities and comorbidities, improving the quality of life for these patients [1]