Ablynx announced that it has submitted a Marketing Authorisation Application to the European Medicines Agency (EMA) for approval of caplacizumab, its 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.
Dr Edwin Moses, CEO of Ablynx, commented:
“As pioneers in the treatment of aTTP, we are committed to making caplacizumab available to patients suffering from this severe disease for which there is currently no specifically approved drug available. This is a very important moment in the development of Ablynx, as we prepare to commercialise our first product and become a fully vertically integrated biopharmaceutical company. We look forward to working with the EMA during this review process.”
