Caplacizumab

Bivalent Single Domain Antibody (nanobody)Rx: PrescriptionCompound: Approved

Also known as: ALX-0681, ALX‑0681, anti-vWF nanobody, Cablivi, Caplacizumab‑yhdp

Educational Only — Not medical advice. Consult a qualified clinician before using any peptide.

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Summary

Caplacizumab is a humanized nanobody approved for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppression.

Mechanism of Action

Binds the A1 domain of von Willebrand factor, blocking its interaction with platelet glycoprotein Ib and preventing microvascular platelet aggregation.

Routes of Administration

IntravenousSubcutaneous

Goals & Uses

  • Accelerate platelet count normalizationTherapeuticHigh
  • Prevention of aTTP exacerbation and recurrenceHematologyHigh
  • Organ damage prevention (renal, neurological)End Organ ProtectionModerate
  • Reduction of microvascular thrombosisAntithromboticHigh
  • Prevent aTTP relapseTherapeuticHigh
  • Treatment of acquired thrombotic thrombocytopenic purpura (aTTP)Hematology / Thrombotic DisorderHigh

Contraindications

  • Active clinically significant bleedingHematologicHigh
  • Hypersensitivity to caplacizumab or excipientsAllergy / ImmunologicHigh

Adverse Effects

  • Hypersensitivity / anaphylaxisImmunologicalRare
  • Injection site reactionsLocalCommon
  • HeadacheNeurologicCommonPain in the head or upper neck
  • Bleeding / hemorrhageHematologicCommon
  • PyrexiaGeneralUncommon
  • FatigueGeneralCommonLow energy or tiredness
  • Injection site reactionLocalCommonRedness, swelling, itching, bruising, or pain at the injection site
  • Bleeding (e.g., epistaxis, gingival bleeding)HematologicCommon

Drug Interactions

  • Antiplatelet agents (e.g., aspirin, clopidogrel)Moderate
  • Anticoagulants (e.g., heparin, warfarin, DOACs)High
  • Thrombolytics (e.g., alteplase)High
  • Anticoagulants (e.g., warfarin, heparin)Moderate

Population Constraints

  • PregnancyReproductive SafetyRelative
  • Pediatric patients (under 18 years)AgeRelative
  • Severe hepatic impairmentOrgan ImpairmentRelative
  • BreastfeedingReproductiveRelative
  • Pediatric patients <12 yearsAgeAbsolute

Regulatory Status

  • European UnionApprovedApproved: Acquired thrombotic thrombocytopenic purpura (aTTP) in adults and adolescents ≥12 yearsEMA approval 2019.
  • United StatesApprovedApproved: Acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressionFDA approval 2019; marketed as Cablivi.
  • United KingdomApprovedApproved: Treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapyApproved by MHRA following EU approval; NICE has issued guidance supporting use.

Approved by FDA (US) and EMA (EU) for adult and adolescent (≥12 y) aTTP; marketed under the brand name Cablivi.

Evidence & Sources

No sources recorded yet.

Frequently Asked Questions

What is Caplacizumab?

Caplacizumab is a humanized nanobody approved for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppression.

What is Caplacizumab used for?

Caplacizumab is educationally associated with: Accelerate platelet count normalization, Prevention of aTTP exacerbation and recurrence, Organ damage prevention (renal, neurological), Reduction of microvascular thrombosis, Prevent aTTP relapse, Treatment of acquired thrombotic thrombocytopenic purpura (aTTP). Educational only — not medical advice.

How is Caplacizumab administered?

Recorded routes of administration: Intravenous, Subcutaneous.

What are the potential side effects of Caplacizumab?

Reported adverse effects include: Hypersensitivity / anaphylaxis, Injection site reactions, Headache, Bleeding / hemorrhage, Pyrexia, Fatigue, Injection site reaction, Bleeding (e.g., epistaxis, gingival bleeding). This list is not exhaustive — consult a qualified clinician.

Who should avoid Caplacizumab?

Recorded contraindications: Active clinically significant bleeding, Hypersensitivity to caplacizumab or excipients. Consult a qualified clinician before use.