NCT05982938

Danicopan Early Access Program

Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria
• PNH
• Extravascular Hemolysis

Interventions

Type	Name	Description
DRUG	Danicopan	Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.

Timeline

• First posted: 2023-08-09
• Last updated: 2026-03-06