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CABLIVI^® (caplacizumab-yhdp) is the first and only therapy targeted to prevent microthrombi in adults with aTTP/iTTP^1,2

Each aTTP/iTTP episode can be unpredictable with microthrombi-driven risks^3-5

MOD

CABLIVI is the first and only therapy targeted to block microthrombi in adults with aTTP/iTTP^1,2

CABLIVI rapidly neutralizes vWF activity, suppressing platelet adhesion from day 1 and throughout treatment^9,10

CABLIVI MOA: The first of its kind

Acquired thrombotic thrombocytopenic purpura, or aTTP, is a rare, rapidly progressing, life-threatening autoimmune disease. Left untreated, up to 90% of aTTP patients die.

Historical treatment includes plasma exchange and immunosuppressive therapy, which has improved prognosis. However, despite these treatments, the mortality rate remains high at 8 to 20%. In a large retrospective study, the median time from diagnosis to death was 9 days despite treatment with plasma exchange.

Symptoms of aTTP can be complex and variable. Most common signs include thrombocytopenia, microangiopathic hemolytic anemia, and organ ischemia, resulting from microvascular occlusion. Clinical manifestations of organ ischemia can include strokes, transient ischemic attacks, myocardial infarction, and renal and mesenteric ischemia.

aTTP is caused by the severe deficiency of a von Willebrand factor-cleaving protease, ADAMTS13. This deficiency is due to formation of autoantibodies directed against ADAMTS13. Normally, ADAMTS13 breaks apart ultra-large strings of von Willebrand factor, known as vWF, into smaller pieces. In aTTP patients, however, ADAMTS13 is blocked by autoantibodies, thereby greatly increasing the amount of ultra-large vWF in circulation. Thousands of circulating platelets bind to these ultra-large vWF strings, forming platelet-rich blood clots within small blood vessels throughout the body. These small clots are known as microthrombi.

As of February 2019, CABLIVI was approved by the FDA for aTTP in combination with plasma exchange and immunosuppressive therapy. Plasma exchange removes vWF and autoantibodies and replenishes ADAMTS13. Immunosuppressive therapies are intended to inhibit anti-ADAMTS13 autoantibody production. Neither directly affects the binding of platelets to vWF.

CABLIVI is a monoclonal antibody fragment that was developed to inhibit microthrombi formation in aTTP by binding specifically to the A1 domain of vWF. CABLIVI blocks the interaction between vWF and platelets, thereby reducing both vWF-mediated platelet adhesion and platelet consumption. This inhibits the formation of microthrombi while the underlying autoimmune disease is treated with immunosuppressive therapy.

Choose CABLIVI upon clinical suspicion of aTTP/iTTP and block microthrombi formation

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ISTH Guideline recommendations

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Who should not start CABLIVI?

CABLIVI is contraindicated in patients with a previous severe hypersensitivity reaction to caplacizumab-yhdp or to any of its excipients
Withhold CABLIVI treatment 7 days prior to elective surgery, dental procedures, or other invasive interventions

ADAMTS13=a disintegrin and metalloproteinase with a thrombospondin type 1 motif, 13; aTTP/iTTP=acquired/immune-mediated thrombotic thrombocytopenic purpura; ISTH=International Society on Thrombosis and Haemostasis; MOA=mechanism of action; PEX=plasma exchange; ULvWF=ultra-large von Willebrand factor; vWF=von Willebrand factor.

INDICATION

CABLIVI (caplacizumab-yhdp) 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.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS:
CABLIVI is contraindicated in patients with previous severe hypersensitivity reaction to caplacizumab-yhdp or to any of its excipients. Hypersensitivity reactions have included urticaria.

WARNINGS AND PRECAUTIONS:
Hemorrhage:

CABLIVI increases the risk of bleeding. 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.
Avoid concomitant use of CABLIVI with antiplatelet agents, thrombolytic drugs, heparin or anticoagulants. If clinically significant bleeding occurs, interrupt use of CABLIVI. 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. If emergency surgery is needed, the use of von Willebrand factor concentrate may be considered to correct hemostasis. After the risk of surgical bleeding has resolved, and CABLIVI is resumed, monitor closely for signs of bleeding.

ADVERSE REACTIONS:
In adults, the most common adverse reactions (>15% of patients) are epistaxis, headache, and gingival bleeding. In pediatric patients, the most frequently reported adverse reactions are epistaxis and tachycardia.

DRUG INTERACTIONS:
Concomitant use of CABLIVI with any anticoagulant or antiplatelet agent may increase the risk of bleeding. Avoid concomitant use when possible. Assess and monitor closely for bleeding with concomitant use.

PREGNANCY:
There are no available data on CABLIVI use in pregnant women to inform a drug associated risk of major birth defects and miscarriage.

Fetal/neonatal adverse reactions: CABLIVI may increase the risk of bleeding in the fetus and neonate. Monitor neonates for bleeding.
Maternal adverse reactions: All patients receiving CABLIVI, including pregnant women, are at risk for bleeding. Pregnant women receiving CABLIVI should be carefully monitored for evidence of excessive bleeding.

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Instructions For Use
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INDICATION

IMPORTANT SAFETY INFORMATION

References: 1. CABLIVI. Prescribing information. Sanofi. 2. Scully M, Cataland SR, Peyvandi F, et al; HERCULES Investigators. Caplacizumab treatment for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2019;380(4):335-346. doi:10.1056/NEJMoa1806311 3. Masias C, Wu H, McGookey M, Jay L, Cataland S, Yang S. No major differences in outcomes between the initial and relapse episodes in patients with thrombotic thrombocytopenic purpura: the experience from the Ohio State University Registry. Am J Hematol. 2018;93(3):E73-E75. doi:10.1002/ajh.25002 4. Schieppati F, Russo L, Marchetti M, et al. Low levels of ADAMTS-13 with high anti-ADAMTS-13 antibodies during remission of immune-mediated thrombotic thrombocytopenic purpura highly predict for disease relapse: a multi-institutional study. Am J Hematol. 2020;95(8):953-959. doi:10.1002/ajh.25845 5. Knoebl P, Cataland S, Peyvandi F, et al. Efficacy andsafety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study. J Thromb Haemost. 2019;18(2):479-484. doi:10.1111/jth.14679 6. Joly BS, Coppo P, Veyradier A. Thrombotic thrombocytopenic purpura. Blood. 2017;129(21):2836-2846. doi:10.1182/blood-2016-10-709857 7. Kremer Hovinga JA, Coppo P, Lämmle B, Moake JL, Miyata T,Vanhoorelbeke K. Thrombotic thrombocytopenic purpura. Nat Rev Dis Primers. 2017;3:17020. doi:10.1038/nrdp.2017.20 8. Azoulay E, Bauer PR, Mariotte E, et al; Nine-i Investigators. Expert statement on the ICU management of patients with thrombotic thrombocytopenic purpura. Intensive Care Med. 2019;45(11):1518-1539. doi:10.1007/s00134-019-05736-5 9. Ulrichts H, Silence K, Schoolmeester A, et al. Antithrombotic drug candidate ALX-0081 shows superior preclinical efficacy and safety compared with currently marketed antiplatelet drugs. Blood. 2011;118(3):757-765. doi:10.1182/blood-2010-11-317859 10. Peyvandi F, Scully M, Kremer Hovinga JA, et al; TITAN Investigators. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2016;374(6):511-522. doi:10.1056/NEJMoa1505533

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