New hope for PNH patients: early access to danicopan combo therapy

NCT ID NCT05982938

First seen Jan 06, 2026 · Last updated May 10, 2026 · Updated 28 times

Summary

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant anemia despite current treatment with Soliris or Ultomiris. Participants must be at least 18 years old, have been on Soliris or Ultomiris for 6 months, and meet specific blood count criteria. The goal is to control disease by reducing red blood cell destruction outside blood vessels.

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