Sanofi’s first Ablynx drug, rare blood disorder treatment Cablivi, clears final EU hurdle

Sanofi will soon be able to reap the first sales from its €3.9 billion ($4.5 billion) buyout of Ablynx, as Cablivi, the lead asset behind the deal, has won EU approval.

After a positive opinion from the European Medicines Agency’s committee for medicinal Products for Human Use (CHMP) in June, Sanofi said on Monday that the drug had been cleared to treat European patients with a rare blood-clotting disorder called acquired thrombotic thrombocytopenic purpura (aTTP).

That makes it the first drug approved specifically for the condition, and it also helps the therapy take its first step toward the $500 million in peak sales that analysts have projected. Meanwhile, the FDA has granted the Cablivi application priority review, setting it up for a decision by Feb. 6.

People with aTTP currently need to go through daily plasma exchange and immunosuppression, but it is still linked with aTTP episodes that cause deaths in up to 20% of cases. In a phase 3 study dubbed Hercules, the addition of Cablivi helped cut the time to platelet count response and significantly reduced the recurrences of aTTP by 67% and related deaths by 74%. It also pared down the length of hospitalization and intensive care unit stays.

Given the estimated 7,500 cases of aTTP in North America, Europe and Japan, plus Cablivi’s clear efficacy advantage, Jefferies analysts previously predicted the drug could reach $500 million in peak sales.

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The drug boosts Sanofi’s ambitions of “becoming the leading rare blood disorders company,” said Genzyme head Bill Sibold in a statement. Under that flag, Sanofi bought Biogen spinoff Bioverativ for $11.6 billion and beat Novo Nordisk’s bid for Ablynx on a roll in January.

Cablivi is Ablynx’s first product developed on its nanobody platform to have crossed a regulatory finish line. The company contends that nanobodies have several advantages over other molecules and are ideal building blocks for novel biologics. Besides Cablivi, Ablynx adds to Sanofi a deep pipeline, including an inhalable drug that treats RSV in infants. Readouts from a phase 2b study of that drug are expected later this year.

From the Bioverativ acquisition, Sanofi got hemophilia drugs Eloctate and Alprolix. For the second quarter, Eloctate turned in sales of €176 million, up 20% at constant exchange rates, while Alprolix’s €81 million represented 6.6% growth.

Down in its rare blood disorders pipeline, there’s Sangamo-partnered gene-edited cell therapy BIVV003, whose new drug application in sickle cell disease has been accepted by the FDA. And sutimlimab (formerly BIVV009) is being tested in phase 3 in cold agglutinin disease.