Ablynx n.v. has submitted a Marketing Authorisation Application to the European Medicines Agency for approval of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura.
Ablynx n.v. announced that it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for approval of caplacizumab, its first-in-class anti-von Willebrand factor (vWF) Nanobody for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), an ultra-rare, acute, life-threatening blood clotting disorder with a high unmet medical need.
The MAA includes data from the Phase II TITAN study in patients with aTTP which demonstrated a statistically significant and clinically meaningful benefit of caplacizumab treatment in reducing the time to platelet count normalisation and reducing recurrences while on drug treatment. Results of post-hoc analyses of the Phase II TITAN study further demonstrated that caplacizumab dramatically reduced the number of patients experiencing major thromboembolic events, as compared to placebo. If approved, caplacizumab will be the first therapeutic specifically indicated for the treatment of aTTP.
Comment:The confirmatory Phase III HERCULES study is currently ongoing and will support the BLA filing in the United States. Results from this Phase III study are expected by the end of 2017. Caplacizumab has the potential to be the first therapeutic specifically approved for the treatment of acquired TTP.
Related news and insights
Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH])
Merck KGaA, a leading science and technology company, announced that its two Phase III EVOLUTION clinical trials (evolution RMS 1 and evolution RMS 2) investigating the efficacy and safety of evobrutinib did not meet their primary endpoints of reducing annualized relapse rates (ARR) in people with relapsing multiple sclerosis (RMS) compared to oral teriflunomide (Aubagio) (0.11 vs. 0.11 in evolution RMS 1 and 0.15 for evobrutinib vs. 0.14 for teriflunomide in evolution RMS 2, p=NS in both trials)