Phase III programme for alicaforsen enema in the orphan disease pouchitis

A drug for a rare gut condition that has no approved treatment is about to enter its final stage of human testing. The drug, alicaforsen, targets pouchitis—a painful inflammation that can develop in people who have had their colon removed and a surgically created internal pouch to replace it. There is currently no licensed therapy for this condition, which affects thousands of patients in Europe and the US. Alicaforsen works by blocking a molecule called ICAM-1, which plays a role in the inflammatory process. It has already completed Phase 2 trials and received orphan drug status from regulators on both sides of the Atlantic. The company behind the drug, Atlantic Healthcare, has secured agreement from regulators that a single, small placebo-controlled Phase 3 trial will be enough to apply for marketing approval. If the trial succeeds, patients with pouchitis would for the first time have a specifically approved treatment, rather than relying on off-label or experimental options. The broader inflammatory bowel disease market, of which pouchitis is a part, is worth $3.5 billion annually and growing at 10 percent per year.