Roche's Emicizumab Nears Approval for New Indication in China

Text | Jia Yi

On March 30, the Insight database showed that the review status of Roche’s second indication application for emicizumab (acceptance numbers: JXSS2000012/13/14/15) had changed to “under review,” indicating imminent approval in the coming days.

Based on the prior priority review public notice, the indication pending approval is: routine prophylactic treatment for adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) without inhibitors to coagulation factor VIII.

Emicizumab is a recombinant humanized bispecific antibody developed by Roche. Its mechanism of action involves bridging activated coagulation factor IX and coagulation factor X, thereby restoring the coagulation process in patients with hemophilia A. The drug was approved by the FDA for marketing in November 2017 under the brand name Hemlibra, for the prophylactic treatment of bleeding in patients with hemophilia A who have factor VIII inhibitors.

At launch, emicizumab had a narrow indication scope for rare disease treatment, resulting in modest sales, with global sales totaling CHF 230 million from late 2017 to 2018. In October 2018, the FDA approved an expanded indication for emicizumab to include bleeding prophylaxis in patients with all types of hemophilia A. Subsequently, its sales grew rapidly, reaching CHF 1.38 billion globally in 2019.

In China, emicizumab is the first bispecific antibody approved for marketing. It was approved by the NMPA in November 2018 for routine prophylactic treatment of adults and children with hemophilia A (congenital factor VIII deficiency) who have factor VIII inhibitors (brand name: Hemlibra®).

In May 2020, Roche’s application for expanded indications was accepted by the Center for Drug Evaluation (CDE), and it was included in the priority review and approval process by the CDE in September of the same year. Upon approval of this indication, its sales revenue is expected to increase further. Meanwhile, patients with hemophilia A, whose primary treatment modality is clotting factor replacement therapy, will also gain access to a new and effective prophylactic treatment option.