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)

NCT05982938•Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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.

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.

Key Dates

Last Updated

March 6, 2026

Conditions

Paroxysmal Nocturnal HemoglobinuriaPNHExtravascular Hemolysis

Interventions

Danicopan

DRUG

Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356 clinicaltrials@alexion.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 6, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Danicopan Early Access Program

Inclusion Criteria

Exclusion Criteria

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Home Reported Outcomes in PNH

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