Jaguar Health submits Orphan Drug Designation application to FDA for crofelemer

Jaguar Health announced that Jaguar family company Napo Pharmaceuticals has submitted an Orphan Drug Designation application to the U.S. Food and Drug Administration for crofelemer, the company’s novel plant-based prescription drug, for the symptomatic relief and treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy, from bacterial, viral, and parasitic infections including Vibrio cholerae, the bacterium that causes cholera. Due to the very low incidence and prevalence of cholera in the United States, cholera is an orphan indication in this country. Rare diseases, also referred to as orphan indications, are a core Jaguar development focus. In 2024 and 2025, Jaguar expects results from 5 proof-of-concept studies of crofelemer, the company’s novel plant-based prescription drug, from at least 8 clinical sites, including Cleveland Clinic, on three continents for the rare disease indications of microvillus inclusion disease- an ultrarare congenital diarrheal disorder – and short bowel syndrome. In accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for these debilitating conditions in 2025 while the company pursues approval of crofelemer for SBS and MVID from the European Medicines Agency and the U.S. Food and Drug Administration (FDA). In August 2023 the FDA activated Napo’s Investigational New Drug application for a new crofelemer powder for oral solution formulation for the treatment of MVID.