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CTAP101 Injection

OPKO IP Holdings II, Inc. · Phase 3 active Small molecule

CTAP101 is a recombinant human C1-esterase inhibitor (C1-INH) that regulates complement and contact system activation to prevent hereditary angioedema attacks.

CTAP101 is a recombinant human C1-esterase inhibitor (C1-INH) that regulates complement and contact system activation to prevent hereditary angioedema attacks. Used for Hereditary angioedema (HAE) Types I and II — prophylaxis and acute attack treatment.

At a glance

Generic nameCTAP101 Injection
SponsorOPKO IP Holdings II, Inc.
Drug classC1-esterase inhibitor (C1-INH) replacement therapy
TargetC1-esterase inhibitor (C1-INH); complement and contact system regulation
ModalitySmall molecule
Therapeutic areaImmunology / Rare Genetic Disorders
PhasePhase 3

Mechanism of action

CTAP101 replaces deficient or dysfunctional C1-esterase inhibitor protein, which normally suppresses excessive activation of the complement cascade and bradykinin-generating contact system. By restoring C1-INH levels and activity, the drug prevents the uncontrolled release of vasoactive mediators that cause the characteristic swelling episodes in hereditary angioedema (HAE).

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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