Jaguar Health submitted an amended protocol to the U.S. Food and Drug Administration on November 20 2025 for its ongoing placebo‑controlled clinical trial of crofelemer in pediatric patients with microvillus inclusion disease (MVID). The amendment is designed to support a potential accelerated approval pathway and to position the company for a Breakthrough Therapy designation.
The trial, which is investigator‑initiated, has shown that crofelemer can reduce parenteral support by up to 37% in MVID patients. MVID is an ultrarare genetic disorder affecting roughly 100–200 infants worldwide, and no approved therapies exist. Crofelemer’s plant‑based, anti‑secretory mechanism blocks chloride secretion in the gut, reducing fluid loss and the need for lifelong parenteral nutrition.
Jaguar Health holds orphan drug designation from both the FDA and the European Medicines Agency for crofelemer in MVID and short‑bowel syndrome with intestinal failure (SBS‑IF). The amended protocol will be reviewed in the upcoming FDA cycle, following a meeting with the agency on October 2 2025 that discussed the trial’s design and preliminary data.
CEO Lisa Conte highlighted the convergence of key clinical and regulatory catalysts in 2025, noting that the amended protocol represents a pivotal moment for the company’s rare‑disease pipeline. She emphasized that a successful approval would not only provide the first therapy for MVID but also strengthen Jaguar’s position in the rare‑disease market.
Financially, Jaguar reported Q3 2025 revenue of $3.1 million and a net loss of $9.5 million, a modest revenue increase but continued operating losses. The company’s high debt‑to‑equity ratio underscores the importance of a successful approval to generate sustainable revenue and improve financial stability.
If the FDA approves crofelemer for MVID, it would become the first therapy for this ultrarare condition, opening a new revenue stream for Jaguar Health and potentially accelerating the company’s broader development program for other indications such as cancer‑related diarrhea.
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