Sanofi's Cablivi® (caplacizumab) gains nod in China for rare blood disorder

On November 5, 2025, Sanofi announced that its innovative medicine Caplacizumab for Injection (brand name: Cablivi®) has been formally approved by China's National Medical Products Administration (NMPA).

The approval covers its use in combination with plasma exchange and immunosuppressive therapy for the treatment of adult and adolescent patients (aged 12 years and older, weighing at least 40 kg) with acquired thrombotic thrombocytopenic purpura (aTTP, also known as immune-mediated thrombotic thrombocytopenic purpura, iTTP).

Thrombotic Thrombocytopenic Purpura (TTP) is an acute, life-threatening thrombotic microangiopathy with an annual incidence of 2–6 cases per million people. As an ultra-rare disease, it has been included in China's Second List of Rare Diseases. Immune-mediated TTP (iTTP) is the most common clinical form, accounting for approximately 95% of all TTP cases. Its clinical manifestations include severe bleeding due to thrombocytopenia, microangiopathic hemolytic anemia (MAHA), and neuropsychiatric symptoms such as confusion, headache, and aphasia. Some patients may also experience fever and renal impairment. The disease often has a sudden onset and severe progression, with high mortality rates if not treated promptly, posing a significant threat to patients' lives and health.

The current standard treatment for iTTP primarily relies on plasma exchange and immunosuppressive therapy. However, these approaches present certain limitations, including non-response in some patients and high post-treatment relapse rates.

In recent years, with growing understanding of iTTP pathophysiology, targeted therapies have emerged as a key research focus. Beyond caplacizumab, several other targeted agents are under development, including drugs targeting the complement system (such as eculizumab and satralizumab). The development of these therapies provides iTTP patients with expanded treatment options and opens new avenues for future therapeutic strategies.