Caplacizumab

Caplacizumab-yhdp targets the A1-domain of vWF, and inhibits the interaction between vWF and platelets, thereby reducing both vWF-mediated platelet adhesion and platelet consumption.

• CABLIVI is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. CABLIVI is a von Willebrand factor (vWF)-directed antibody fragment indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

• CABLIVI is contraindicated in patients with a previous severe hypersensitivity reaction to caplacizumab-yhdp or to any of the excipients. Hypersensitivity reactions have included urticaria [see Adverse Reactions (6.1) ] .contraindicated

CABLIVI should be administered upon the initiation of plasma exchange therapy. The recommended dose of CABLIVI is as follows: ( 2.1 ) First day of treatment: 11 mg bolus intravenous injection at least 15 minutes prior to plasma exchange followed by an 11 mg subcutaneous injection after completion of plasma exchange on day 1. Subsequent treatment during daily plasma exchange: 11 mg subcutaneous injection once daily following plasma exchange. Treatment after the plasma exchange period: 11 mg subcutaneous injection once daily for 30 days beyond the last plasma exchange. If after initial treatment course, sign(s) of persistent underlying disease such as suppressed ADAMTS13 activity levels remain present, treatment may be extended for a maximum of 28 days. Discontinue CABLIVI if the patient experiences more than 2 recurrences of aTTP, while on CABLIVI. The first dose should be administered by a healthcare provider as a bolus intravenous injection. Administer subsequent doses subcutaneously in the abdomen. ( 2.3 ) 2.1 Recommended Dose and Schedules CABLIVI should be administered upon initiation of plasma exchange therapy. The recommended dose of CABLIVI is as follows: First day of treatment : 11 mg bolus intravenous injection at least 15 minutes prior to plasma exchange followed by an 11 mg subcutaneous injection after completion of plasma exchange on day 1. Subsequent days of treatment during daily plasma exchange : 11 mg subcutaneous injection once daily following plasma exchange.

Hemorrhage: Serious and fatal bleeding can occur. Risk of bleeding is increased in patients with underlying coagulopathies or on concomitant antiplatelet agents or anticoagulants. If clinically significant bleeding occurs, interrupt treatment. Withhold CABLIVI 7 days prior to elective surgery, dental procedures, or other invasive interventions. ( 5.1 ) 5.1 Hemorrhage CABLIVI increases the risk of bleeding [see Adverse Reactions (6.1) ] . In clinical studies, severe bleeding adverse reactions of epistaxis, gingival bleeding, upper gastrointestinal hemorrhage, and metrorrhagia were each reported in 1% of subjects. Overall, bleeding events occurred in approximately 58% of patients on CABLIVI versus 43% of patients on placebo. In the postmarketing setting, cases of life-threatening and fatal bleeding were reported in patients receiving CABLIVI. The risk of bleeding is increased in patients with underlying coagulopathies (e.g., hemophilia, other coagulation factor deficiencies). It is also increased with concomitant use of CABLIVI with drugs affecting hemostasis and coagulation [see Drug Interactions (7) and Clinical Pharmacology (12.3) ] . Avoid concomitant use of CABLIVI with antiplatelet agents, thrombolytic drugs, heparin, or anticoagulants. Interrupt use of CABLIVI if clinically significant bleeding occurs. If needed, von Willebrand factor concentrate may be administered to rapidly correct hemostasis. If CABLIVI is restarted, monitor closely for signs of bleeding. Withhold CABLIVI for 7 days prior to elective surgery, dental procedures or other invasive interventions.

The following clinically significant adverse reactions are also discussed in other sections of the labeling: Hemorrhage [see Warnings and Precautions (5.1) ] In adults, the most common adverse reactions (incidence >15%) are epistaxis, headache, and gingival bleeding. In pediatric patients, the most frequently reported adverse reactions are epistaxis and tachycardia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ablynx US at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. TITAN and HERCULES The safety of CABLIVI was evaluated in two placebo-controlled clinical studies (HERCULES, in which 71 patients received CABLIVI; and TITAN, in which 35 patients received CABLIVI). The data described below and in the Warnings and Precautions reflect exposure to CABLIVI during the blinded periods of both studies, which include 106 patients with aTTP who received at least one dose, age 18 to 79 years, of whom 69% were female and 73% were White. The median treatment duration with CABLIVI was 35 days (range 1–77 days). The most frequently reported adverse reactions (>15%) were epistaxis, headache and gingival bleeding. Seven patients (7%) in the CABLIVI group experienced an adverse reaction leading to study drug discontinuation.

8.1 Pregnancy Risk Summary There are no available data on CABLIVI use in pregnant women to inform a drug-associated risk of major birth defects and miscarriage. However, there are potential risks of hemorrhage in the mother and fetus associated with use of CABLIVI (see Clinical Considerations ) . In animal reproduction studies, there was no evidence of adverse developmental outcomes with intramuscular administration of caplacizumab-yhdp during organogenesis in guinea pigs at exposures approximately 30 times the AUC in humans at the recommended subcutaneous injection dose of 11 mg (see Data ) . All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. The background rate of major birth defects and miscarriage in the indicated population is unknown. In the U.S. general population, the estimated background rate of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Clinical Considerations Fetal/neonatal adverse reactions CABLIVI may increase the risk of bleeding in the fetus and neonate. Monitor neonates for bleeding [see Warnings and Precautions (5.1) ] . Maternal adverse reactions All patients receiving CABLIVI, including pregnant women, are at risk for bleeding.

Metabolism

Caplacizumab-yhdp pharmacokinetics depends on the expression of the target vWF and are not dose proportional. Higher levels of vWF antigen increase the fraction of drug-target complex retained in the circulation.

In case of overdose, based on the pharmacological action of CABLIVI, there is the potential for an increased risk of bleeding [see Warnings and Precautions (5.1) ] . Monitor patients for signs and symptoms of bleeding if overdose occurs. Consider the use of von Willebrand factor concentrate to correct hemostasis, if needed.

• Feb 6, 2019BLABLA761112Ablynx Nv

1. 1Product Use In Unapproved Indication52232%
2. 2Off Label Use19612%
3. 3Adamts13 Activity Decreased1499.1%
4. 4Platelet Count Decreased1257.6%
5. 5Epistaxis1227.4%
6. 6Fatigue1076.5%
7. 7Contusion835.1%
8. 8Thrombotic Thrombocytopenic Purpura774.7%
9. 9Condition Aggravated764.6%
10. 10Headache764.6%
11. 11Injection Site Pain724.4%
12. 12Adamts13 Activity Abnormal684.2%
13. 13Injection Site Bruising593.6%
14. 14Hospitalisation482.9%
15. 15Platelet Count Abnormal472.9%

The 10 most recently updated of 16 ClinicalTrials.gov registrations naming Caplacizumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP
  Active not recruiting · Observational · 350 enrolled · University of Cologne
  NCT04985318updated 2026-04-30
• Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP
  Terminated · Phase 2 · Interventional · 58 enrolled · University Hospital, Rouen
  NCT04720261updated 2026-02-20
• Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura
  Recruiting · Interventional · 131 enrolled · University Hospital, Rouen
  NCT06291025updated 2026-02-17
• Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
  Completed · Phase 3 · Interventional · 51 enrolled · Sanofi
  NCT05468320updated 2025-12-30
• A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
  Completed · Phase 2 · Phase 3 · Interventional · 21 enrolled · Sanofi
  NCT04074187updated 2025-09-22
• Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura
  Completed · Observational · 223 enrolled · University of Cologne
  NCT05876221updated 2025-09-12
• Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors
  Not yet recruiting · Observational · 116 enrolled · US Thrombotic Microangiopathy Alliance
  NCT06358703updated 2024-11-13
• Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
  Completed · Phase 3 · Interventional · 145 enrolled · Ablynx, a Sanofi company
  NCT02553317updated 2023-04-05
• Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
  Completed · Phase 2 · Interventional · 75 enrolled · Ablynx, a Sanofi company
  NCT01151423updated 2023-04-04
• Comparative Study of ALX-0081 Versus GPIIb/IIIa Inhibitor in High Risk Percutaneous Coronary Intervention (PCI) Patients
  Completed · Phase 2 · Interventional · 364 enrolled · Ablynx, a Sanofi company
  NCT01020383updated 2023-04-04

Frequently asked questions

How does Caplacizumab work?

Caplacizumab-yhdp targets the A1-domain of vWF, and inhibits the interaction between vWF and platelets, thereby reducing both vWF-mediated platelet adhesion and platelet consumption.

What is Caplacizumab used for?

According to FDA labeling, Caplacizumab carries indications including: CABLIVI is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. CABLIVI is a von Willebrand factor (vWF)-directed antibody fragment indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Caplacizumab?

Caplacizumab is classified as Other antithrombotic agents, Antibody-Receptor Interactions, Decreased Platelet Aggregation.

What are the brand names for Caplacizumab?

Caplacizumab is marketed under brand names including Cablivi.

What are the contraindications for Caplacizumab?

Caplacizumab labeling lists contraindications including: CABLIVI is contraindicated in patients with a previous severe hypersensitivity reaction to caplacizumab-yhdp or to any of the excipients. Hypersensitivity reactions have included urticaria [see Adverse Reactions (6.1) ] .. Always consult the full prescribing information and a clinician.