Caplacizumab Update

July 2019: Cablivi will shortly be reviewed (by CADTH and INESSS), and recommendations made to Canadian decision makers regarding approval for use and funding. All those diagnosed with TTP are encouraged to help provide the patient perspective to aid decision makers by taking part in the upcoming community survey. The survey will be circulated to the international community and a link will be posted to our website in the next couple of weeks.
 
Cablivi® (caplacizumab-yhdp) is the first medicine approved in the U.S. specifically for the treatment of aTTP. The drug was studied in a multicentre, randomized, double-blind, placebo-controlled Phase 3 clinical study known as HERCULES. Study conclusions were published in the New England Journal of Medicine in January 2019. "Conclusions: Among patients with TTP, treatment with caplacizumab was associated with faster normalization of the platelet count; a lower incidence of a composite of TTP-related death, recurrence of TTP, or a thromboembolic event during the treatment period; and a lower rate of recurrence of TTP during the trial than placebo"---source: New England Journal of Medicine.

Feb 6, 2019: The U.S. Food and Drug Administration (FDA) has approved Cablivi® (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults.  Cablivi is the first FDA approved therapy specifically indicated for the treatment of aTTP. Full press release here.

January 9, 2019: New England Journal of Medicine publishes results of caplacizumab double-blind, controlled trial of 145 patients with TTP.  "CONCLUSIONS: Among patients with TTP, treatment with caplacizumab was associated with faster normalization of the platelet count; a lower incidence of a composite of TTP-related death, recurrence of TTP, or a thromboembolic event during the treatment period; and a lower rate of recurrence of TTP during the trial than placebo". Journal article link here.

October 2, 2017 ABLYNX "announced positive topline results from the Phase III HERCULES study with caplacizumab, the Company's anti-von Willebrand factor (vWF) Nanobody® being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP)." View the full article.

July 26, 2017: Ablynx announced "that the Food and Drug Administration (FDA) has granted Fast Track designation for caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP)." SOURCE: http://www.empr.com/drugs-in-the-pipeline/caplacizumab-attp-autoimmune-b....View the full article.