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Jaguar Health Announces Preliminary Discussion with FDA to Evaluate Breakthrough Therapy Designation for Crofelemer for Treatment of Microvillus Inclusion Disease (MVID)
Pediatric patients with Intestinal Failure (IF) due to MVID, an ultrarare congenital disorder with lethal natural history, that has no available therapies and
About this update from Jaguar Health, Inc.
[{"type":"text","content":"Pediatric patients with Intestinal Failure (IF) due to MVID, an ultrarare congenital disorder with lethal natural history, that has no available therapies and needs lifelong total Parenteral Support (PS), could benefit from expedited development of oral crofelemer via Breakthrough Therapy DesignationCrofelemer has demonstrated meaningful reductions in weekly PS needs in two pediatric MVID patients with initial results to be presented at ESPGHAN 2026 SAN FRANCISCO, CA / ACCESS Newswire / May 6, 2026 / Jaguar Health, Inc. (NASDAQ:JAGX) (\"Jaguar\") family company Napo Pharmaceuticals Inc., announced that the Company recently held a preliminary discussion with the Division of Gastroenterology at U.S. Food and Drug Administration (FDA) to evaluate the possibility of a Breakthrough Therapy Designation (BTD) for oral liquid crofelemer as an adjunctive therapy to PS for the treatment of pediatric patients with intestinal failure (IF) due to microvillus inclusion disease (MVID), an ultrarare congenital disorder with a lethal natural history requiring lifelong PS. Crofelemer has demonstrated preliminary clinical proof of concept (POC) in pediatric MVID patients in two open-label studies, with meaningful reductions in weekly PS requirements for the two MVID patients. Initial results for long-term evidence of efficacy and safety from the open label studies in pediatric MVID patients have been accepted for presentations at the upcoming 58th Annual ESPGHAN (European Society for Paediatric Gastroenterology Hepatology and Nutrition) Meeting in June, 2026. Crofelemer is also being evaluated in a pivotal randomized double-blind placebo-controlled clinical study in pediatric MVID subjects which has completed enrollment in this ongoing adequate and well-controlled trial.\"We are pleased with the initial clinical POC results for the safety and effectiveness of liquid oral crofelemer as adjunctive therapy to life sustaining PS in pediatric IF patients. Our objective is to evaluate expedited pathways to support availability of crofelemer for the serious unmet need of IF in patients with MVID. There are no available therapies for MVID other than lifelong PS which is inadequate and associated with profound comorbidities. Breakthrough therapy designation in the US and PRIME in the European Union are important potential regulatory pathways to expedite ac...