Jaguar Health granted Orphan Drug designation for crofelemer for diarrhea

Jaguar Health’s (JAGX) Napo Pharmaceuticals announced that the U.S. Food and Drug Administration has granted orphan-drug designation to crofelemer, the company’s novel plant-based prescription drug, for treatment of diarrhea in cholera. Due to the very low incidence and prevalence of cholera in the United States, cholera is an orphan indication in this country. Crofelemer is also the subject of five other rare/orphan disease-related clinical efforts – three investigator-initiated trial proof-of-concept studies and two Phase 2 studies – for the indications of short bowel syndrome with intestinal failure and/or microvillus inclusion disease in the US, European Union, and/or Middle East/North Africa regions. Dosing of the first patient in each of these five studies is expected to occur throughout December 2024 and Q1 2025, with availability of IIT proof-of-concept results potentially in Q2 2025. In accordance with the guidelines of specific EU countries, published data from clinical investigations in SBS-IF and MVID could support reimbursed early patient access to crofelemer for these debilitating conditions in those countries. Crofelemer has also been granted ODD by the FDA and the EMA for both MVID and SBS. Additionally, Jaguar intends to pursue orphan-drug designation and a Tropical Disease Priority Review Voucher for the indication of treatment of diarrhea in cholera with a proprietary second-generation anti-secretory agent, NP-300, which, like crofelemer, is sustainably derived from the Croton lechleri tree.