Ablynx files blood disorder drug in EU

Ablynx has filed a first-in-class drug for an ultra-rare, life threatening blood clotting disorder with European regulators.

The Belgian biotech filed caplacizumab with the European Medicines Agency for treatment of acquired thrombotic thrombocytopenic purpura (aTTP), an acute disorder for which treatment options are limited.

If approved, the drug will be the company’s first marketed drug.

Caplacizumab is a first-in-class anti-von Willebrand factor “nanobody” – Ablynx’s proprietary therapeutic proteins based on single-doman antibody fragments combining advantages of antibody drugs with some features of small-molecule drugs.

The marketing authorisation application includes data from the phase 2 TITAN study in patients with aTTP, demonstrating 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.

Ablynx is due to report results of the phase 3 HERCULES study in the second half of 2017, which will support a US filing in 2018.

Post-hoc analyses of the study demonstrated caplacizumab reduced the number of patients experiencing major thromboembolic events, compared with placebo.


If approved, caplacizumab will be the first therapeutic specifically indicated for the treatment of aTTP.

Ablynx intends to market caplacizumab in Europe and North America.

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.”

In October, Ablynx suffered a setback after AbbVie decided not to take an option to develop rheumatoid arthritis candidate drug, vobarilizumab, just as phase 3 trials were about to begin.

AbbVie was expected to take the option to develop vobarilizumab as its Humira, which has a sizeable chunk of the RA market, is due to lose patent protection in the coming years in the US and EU.

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