immunodeficiency 55

ORPHA: 5052272 Treatments Available

Overview

human disease

Available Treatments (2)

DrugFormStatusCountriesLead Time
Berotralstat
Orphan
CapsuleFDA Approved414d
C1 esterase inhibitor (human)
Orphan Cold Chain
Lyophilized powder for reconstitutionFDA Approved47d

Classification & Codes

Orphanet Code

ORPHA:505227
immunodeficiency 55
OrphanetORPHA:505227
Treatments2 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO