Napo Pharmaceuticals Discusses Breakthrough Therapy Designation with FDA
Jaguar Health family company Napo Pharmaceuticals announced that the company recently held a preliminary discussion with the Division of Gastroenterology at U.S. Food and Drug Administration to evaluate the possibility of a Breakthrough Therapy Designation for oral liquid crofelemer as an adjunctive therapy to PS for the treatment of pediatric patients with intestinal failure due to microvillus inclusion disease, an ultrarare congenital disorder with a lethal natural history requiring lifelong PS. Crofelemer has demonstrated preliminary clinical proof of concept 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 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 access. To the best of our knowledge, crofelemer is being used to treat approximately 4% of the estimated living pediatric MVID patients. Crofelemer has demonstrated meaningful reductions in PS needs for pediatric IF patients with MVID. 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. Since PS is associated with significant comorbidities, reductions in PS needs are expected to improve clinical outcomes as well as quality of life measures," said Dr. Pravin Chaturvedi, Napo's Chief Scientific Officer and Chair of the company's Scientific Advisory Board. Jaguar family company, Napo Pharmaceuticals, Inc., discussed the possibility of Breakthrough Therapy Designation for crofelemer for treatment of MVID as adjunctive therapy to lifelong PS with the FDA, given the preliminary clinical POC results in the open label studies. Breakthrough Therapy designation is an FDA program intended to expedite the development and review of drugs that are being developed to treat serious or life-threatening diseases or conditions where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints.
