Sanofi scores FDA nod for $270K nanobody drug Cablivi, its first from the Ablynx deal

Sanofi scooped up nanobody biotech Ablynx last year in a $4.5 billion deal—and now, the buyout may be starting to pay off. Cablivi, an Ablynx nanobody for a rare blood-clotting disorder, has snagged its FDA green light.

Cablivi scored its FDA nod in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura, or aTTP. The drug will be available late in the first quarter and cost $270,000 before rebates and discounts to treat a typical episode of aTTP, Sanofi said.

Sanofi figures that Cablivi's improvement in patient outcomes will be attractive to patients and payers, and it sees the drug as a building block in a new blood disorder franchise. Analysts have said it could hit $500 million in peak sales worldwide. In a clinical study, the drug combined with plasma exchange and immunosuppression significantly bested standard of care in reducing patients' platelet count response time, aTTP-related death, aTTP recurrence or a major thromboembolic event during treatment, the drugmaker reported.

In an interview, Sanofi Genzyme global head of medical affairs Rand Sutherland said patients with the disease form abnormal blood clots through a process that Cablivi “will ultimately target and modify.” The disease is challenging to diagnose and once diagnosed requires “very aggressive” treatment, Sutherland said. The standard of care has involved a plasma exchange, plus corticosteroids and immunomodulators, which Sutherland called an “invasive and intensive” process that often occurs in intensive care units.  

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Before plasma exchanges, the mortality rate for patients was about 90%, Sutherland said. Even now, the mortality rate is about 20%, according to the Genzyme executive, which he said is not an "acceptable standard of care." Cablivi’s goal is to conduct a process similar to a plasma exchange inside a patient’s body “essentially on a daily basis,” Sutherland said. 

Aside from being the first drug approved for aTTP, Cablivi is also Sanofi’s first nanobody approved by the FDA. The drugmaker picked up the technology in its Ablynx acquisition last year, inked in part to help beef up in rare diseases. 

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Nanobodies are “small fragments of antibodies” that researchers found in the Camelidae family of animals that includes llamas and camels, Sutherland said. Humans have larger and more complicated antibodies, Sutherland said, while antibodies in the animals are smaller. Nanobodies make "ideal building blocks" for biologic drugs against a variety of diseases, Ablynx says.

Aside from its Ablynx buyout, Sanofi also picked up Bioverativ last year for $11.6 billion in a push into blood disorders. That deal gave the drugmaker a presence in hemophilia.