EMA, New Products

Sanofi reaps first fruit of Ablynx buyout with a European OK for its lead rare disease drug

Just weeks after closing its $4.8 billion acquisition of Ablynx, Sanofi has bagged a European approval for its lead drug, caplacizumab, which treats an ultra-rare blood clotting disease.

The OK — based on a pair of Phase II and Phase III studies — marks the first regulatory validation for Ablynx’s platform tech for small “nanobodies” that are a sliver of the size of regular antibodies.

Caplacizumab treats acquired thrombotic thrombocytopenic purpura (aTTP), a disorder characterized by blood clots in small blood vessels that triggers low platelet counts. Researchers found that the drug significantly reduced the time it took for the platelet counts to return to normal and lowered the the percentage of patients who died or saw the disease recur.

Bill Sibold

Sanofi believes its treatment will add to the current standard of care, which consists of daily plasma exchange and immunosuppression. About 20% of patients receiving such treatment do not survive an episode of aTTP, with most of the deaths occurring within 30 days of diagnosis.

Instead of targeting the enzyme that’s responsible for cleaving the large protein involved in clotting — the von Willebrand factor (vWf) — caplacizumab blocks the binding process between the vWfs with platelets.

“This approval is the next step towards our goal of becoming the leading rare blood disorders company in the industry,” said Bill Sibold, EVP and head of Sanofi Genzyme, in a statement.

Sanofi dove into the hemophilia market by striking an $11.6 billion Bioverativ buyout, days before announcing the Ablynx deal. The Biogen spinout is also developing a number of treatments for other blood disorders, including cold agglutinin disease, beta thalassemia and sickle cell disease.

Sanofi Genzyme, the French drugmaker’s rare disease unit, will be marketing the caplacizumab as Cablivi.

An FDA approval is expected to follow in February 2019. Jefferies’ Peter Welford previously estimated peak sales at $400 million in the two markets combined.


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