The European Commission (EC) has granted marketing authorisation for Sanofi’s Cablivi (caplacizumab) indicated to treat adults with acquired thrombotic thrombocytopenic purpura (aTTP) episodes.
Cablivi is said to be the first medicine specifically approved for the treatment of aTTP, a rare, life-threatening, autoimmune-based blood clotting disorder.
Its symptoms include extensive formation of clots in small blood vessels, which can result in severe thrombocytopenia, microangiopathic hemolytic anaemia, ischemia and organ damage.
Developed by Sanofi’s subsidiary Ablynx, Cablivi is a nanobody-based drug and is under the Sanofi Genzyme’s Rare Blood Disorders franchise.
Sanofi Genzyme executive vice-president and head Bill Sibold said: “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.
“This approval is the next step towards our goal of becoming the leading rare blood disorders company in the industry.”
The EC has reviewed data from phase II TITAN and phase III HERCULES clinical trials conducted to assess the safety and efficacy of Sanofi’s Cablivi in a total of 220 aTTP patients.
Cablivi was given in conjunction with standard-of-care treatment, daily plasma exchange (PEX) and immunosuppression.
In the HERCULES study, caplacizumab in combination with the standard-of-care met the primary endpoint of a significantly shorter time to platelet count response.
The combination also demonstrated a significant decrease in disease-related death and recurrence. It also facilitated a clinically meaningful reduction in PEX use and hospital stay, compared with the placebo.
In clinical trials, caplacizumab is said to have showed a safety profile consistent with its mechanism of action. The most frequent adverse reactions were epistaxis, headache and gingival bleeding.
In the TITAN and HERCULES trials, no caplacizumab-related deaths were reported.
Also, the US Food and Drug Administration (FDA) has accepted the biologics license application of Sanofi’s Cablivi for priority review to treat aTTP patients aged 18 years and above.