Belgium-based Ablynx has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for approval of caplacizumab anti-von Willebrand factor (vWF) Nanobody to treat acquired thrombotic thrombocytopenic purpura (aTTP).

aTTP is an ultra-rare, acute, life-threatening blood clotting disorder with a high unmet medical need.

It has a sudden onset caused by impaired activity of the ADAMTS13 enzyme, leaving ULvWF molecules uncleaved.

The molecules bind to blood platelets spontaneously and result in severe thrombocytopenia and micro-clot formation in small blood vessels throughout the body.

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In the MAA, the company provided data from the Phase II TITAN study carried out in patients with aTTP, which demonstrated a benefit of caplacizumab treatment in reducing the time to platelet count normalisation and reducing recurrences while on drug treatment.

Caplacizumab will be the first therapeutic specifically indicated for the treatment of aTTP in the case of its approval.

"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."

Ablynx CEO Dr Edwin Moses said: “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.”

Caplacizumab received orphan drug designation in Europe and the US in 2009 and blocks the interaction of ultra-large vWF multimers (ULvWF) with platelets.

The company evaluated the efficacy and safety of caplacizumab in conjunction with the standard of care of plasma exchange (PEX) and immunosuppression in the Phase II TITAN study within 75 patients with aTTP.

Image: Caplacizumab – Ablynx's anti-vWF Nanobody. Photo: © Ablynx.