Sanofi announced that the Food and Drug Administration (FDA) has approved Cablivi (caplacizumab-yhdp) for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

Cablivi, a novel Nanobody-based agent, works by inhibiting the interaction between von Willebrand factor (vWF) and platelets, thereby reducing both vWF-mediated platelet adhesion and platelet consumption. Nanobodies are a novel class of therapeutic proteins based on single-domain antibody fragments that contain the unique structural and functional properties of naturally-occurring heavy chain only antibodies.

The FDA approval was supported by data from the Phase 3 HERCULES study (N=145) which evaluated Cablivi vs placebo in combination with plasma exchange and immunosuppressive therapy (current standard of care) in adults with aTTP.

Results showed that patients in the Cablivi arm experienced a significantly shorter time to platelet count response (platelet count ≥150,000/µL followed by cessation of daily plasma exchange within 5 days) vs patients in the plasma exchange and immunosuppression arm alone (hazard ratio [HR] 1.55, 95% CI, 1.10, 2.20; P =.01), the study’s primary efficacy endpoint.

Treatment with Cablivi also resulted in a significant reduction on a composite endpoint of aTTP-related death, aTTP recurrence, or a major thromboembolic event during treatment vs plasma exchange and immunosuppression alone (12.7% vs 49.3%; P <.0001), as well as a significantly lower percentage of aTTP recurrences in the overall study period (13% vs 38%; P <.001).

Epistaxis, headache, and gingival bleeding were the most commonly reported adverse events in clinical trials.

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“Cablivi is the first targeted treatment that inhibits the formation of blood clots,” stated Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s CDER. “It provides a new treatment option for patients that may reduce recurrences.”

Cablivi for injection will be supplied as an 11mg preservative-free lyophilized powder in single-dose vials. It is anticipated to launch sometime in the first quarter of 2019.

For more information call (800) 745-4447 or visit  Sanofi.us.