Sanofi blood disorder drug wins FDA approval

(Reuters) – The U.S. Food and Drug Administration (FDA) said on Wednesday it had approved Sanofi SA’s drug to treat a rare blood-clotting disorder in adults in combination with standard-of-care treatments.

The condition, called acquired thrombotic thrombocytopenic purpura (aTTP), is a life-threatening, autoimmune disorder characterized by clot formation in small blood vessels throughout the body, cutting off blood supply to various body parts and potentially causing strokes and heart attacks.

The company said earlier that despite the current standard-of-care treatments – daily plasma exchange and immunosuppression – about 20 percent of the aTTP patients still die, often within 30 days of diagnosis.

“Cablivi is the first targeted treatment that inhibits the formation of blood clots,” Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said. “It provides a new treatment option for patients that may reduce recurrences.”

Cablivi is already approved in the European Union as a treatment for acquired forms of the disorder, called thrombotic thrombocytopenic purpura.

It should come with a warning about the risk of severe bleeding, the FDA said.

Cablivi was originally developed by Ablynx, a Belgium biotech firm that Sanofi acquired in January last year for 3.9 billion euros ($4.53 billion) right after it had bagged U.S. haemophilia specialist Bioverativ for $11.6 billion.

Caplacizumab, the active substance in Cablivi, is a nanobody that acts by blocking platelet aggregation which can form clots in blood vessels. Before it was acquired by Sanofi, Ablynx had forecast peak caplacizumab sales of 1.2 billion euros.

Cablivi, which received European approval in September last year, is marketed in Germany and is being rolled out in other European countries.