Status:
AVAILABLE
Danicopan Early Access Program
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
PNH
Eligibility:
All Genders
18+ years
Brief 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 ...
Eligibility Criteria
Inclusion
- Key Inclusion Criteria
- Male or female, aged 18 years and older
- Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L.
- Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.
- Platelet count ≥ 30,000/µL without the need for platelet transfusions.
- Absolute neutrophil counts ≥ 500/µL.
- Vaccinated against N meningitidis.
- Key Exclusion Criteria
- Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).
- Laboratory abnormalities at screening, as follows:
- Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN)
- Direct bilirubin \> 2 × ULN, with the exception of:
- participants with increased bilirubin due to EVH in the opinion of the Treating Physician or
- participants with documented Gilbert's syndrome
- Active bacterial or viral infection, a body temperature \> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.
Exclusion
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT05982938
Last Update
November 26 2025
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