Sanofi has EMA approval for its acquired thrombotic thrombocytopenic purpura (aTTP) therapy Cablivi, the main asset behind its €9.3bn acquisition of Belgian biotech Ablynx in January.
The EU regulator has cleared Cablivi (caplacizumab) for adults with aTTP, and Sanofi says that the drug is the first in Europe to be approved specifically for the rare blood-clotting disorder, which causes extensive clot formation in small blood vessels around the body, starving organs of oxygen. The company is also due to hear from the US FDA on its application by 6 February.
At the moment, people with aTTP have to be treated with daily plasma exchange (PEX) and immunosuppressive drugs, and are still a high risk of serious breakthrough attacks that can be fatal in around 20% of cases. In the phase III HERCULES trial, treatment with Cablivi resulted in a significantly shorter time to platelet count response – the primary endpoint – as well as a significant reduction in aTTP-related deaths compared to PEX and immunosuppression.
There’s no word on the cost of the medicine just yet, but analysts at Jefferies have previously suggested that if the price is pitched right Cablivi could be a game-changer in aTTP treatment and generate sales upwards of $500m a year. Prior to its takeover, Ablynx had been suggesting sales of more than €1bn were possible at peak.
Sanofi reckons there are around 7,500 patients with aTTP across the US, the EU and Japan. It has indicated it intends to launch Cablivi in its first EU market – likely Germany – later this year.
The new drug is the first new product to come from Sanofi’s push into rare disease therapies and specifically rare blood disorders, which was also behind its $11.6bn acquisition of Bioverativ earlier this year which added two marketed haemophilia drugs as well as a pipeline of blood disorder products including genome editing drugs and gene therapies.
“The approval of Cablivi provides new hope for people diagnosed with aTTP, who to date have faced a very difficult disease with limited treatment options,” commented Bill Sibold, head of Sanofi’s Genzyme rare disease unit.
“This approval is the next step towards our goal of becoming the leading rare blood disorders company in the industry.”