Amazon teams up with rare disease researchers
A drug derived from an Amazon rainforest tree may become the first approved treatment for two rare intestinal diseases, the CEO of the company developing it stated.
Crofelemer, a plant-based compound extracted from the sap of the Croton lechleri tree, is undergoing clinical trials for microvillus inclusion disease (MVID) and short bowel syndrome with intestinal failure (SBS-IF). Both conditions force patients to rely on intravenous nutrition for up to 20 hours daily, with no approved therapies for MVID and only limited options for a subset of SBS-IF patients.
A demanding lifeline
Patients with intestinal failure depend on total parenteral nutrition (TPN), a lifesaving but demanding regimen of fluids and nutrients delivered directly into the bloodstream. The burden is particularly severe for infants and young children.
“Many require parenteral support up to seven days a week,” said Massimo Radaelli, president and CEO of Napo Therapeutics, the Milan-based firm leading development in Europe. “The side effects resemble some toxicities associated with chemotherapy—serious infections, liver and kidney function problems, metabolic complications.”
The risks are clear. Without effective treatment, prolonged parenteral support use can be deadly. Crofelemer, taken as an oral powder, seeks to reduce reliance on intravenous support by improving intestinal function.
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Early results and a race for approval
An independent study in the United Arab Emirates has monitored pediatric intestinal failure patients treated with crofelemer for over a year. Initial findings, shared in November 2025, demonstrated disease progression modification with crofelemer through reduction of parenteral support that ranged from 12 to 37 percent.
A larger, placebo-controlled trial in pediatric MVID patients is in progress, with results expected in the second quarter of 2026. If successful, the company will pursue Breakthrough Therapy designation from the FDA and the European Medicines Agency’s PRIME programme for MVID to accelerate approval.
MVID is an ultra-rare disease for which no approved treatments currently exist. The immediate focus remains MVID, given its potential for faster regulatory pathways.
The drug is still experimental. Approval would provide the first therapy for MVID, a condition that currently has no treatment options.
The journey from rainforest to medicine is complex. For patients and families enduring daily struggles with intestinal failure, the prospect of a less invasive solution offers meaningful hope. Rare resources like this botanical compound highlight the value of preserving natural ecosystems.
