New hope for rare blood and kidney diseases: iptacopan access program opens
NCT ID NCT05222412
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exhausted all available therapies. The goal is to offer a potential treatment when no satisfactory alternative exists.
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Conditions
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