New Data for Genentech’s Hemlibra (emicizumab-kxwh) Reinforce Safety Profile in People With Hemophilia A

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced results from the final analysis of the Phase IIIb STASEY study, which confirm the favorable safety profile of Hemlibra (emicizumab-kxwh), consistent with the Phase III HAVEN clinical program. In the analysis, no new safety signals were identified with longer-term Hemlibra treatment in adults and adolescents with hemophilia A with inhibitors to factor VIII, the clotting protein that is missing or defective in people with hemophilia A. The data were presented at the virtual International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress, July 17-21.

“As the treatment landscape evolves, determining the long-term benefit/risk profile of medicines for people living with hemophilia A remains a top priority for the community,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “These results provide further confidence in Hemlibra’s favorable safety profile in people with hemophilia A with factor VIII inhibitors, who have historically faced significant treatment challenges.”

Nearly one in three people with severe hemophilia A develop factor VIII inhibitors, antibodies that bind to and block the efficacy of replacement factor VIII. People with hemophilia A with factor VIII inhibitors are at greater risk of more frequent bleeding, including life-threatening bleeds, and may face greater challenges in their day-to-day lives than people with hemophilia who do not have factor VIII inhibitors. Hemlibra has been approved in more than 100 countries worldwide for the treatment of people with hemophilia A with factor VIII inhibitors.

The final analysis of the STASEY study included data from 193 people with hemophilia A with factor VIII inhibitors who received Hemlibra prophylaxis once-weekly for up to two years (median treatment duration of 103.1 weeks). The analysis did not show any new cases of thrombotic microangiopathy or serious thrombotic events (adverse events [AEs] that have been observed in people with bleeding disorders) related to Hemlibra. The most common AEs occurring in 10% or more of people in the STASEY study were joint pain (arthralgia; 17.1%), common cold symptoms (nasopharyngitis; 15.5%), headache (15.0%), injection site reaction (ISR) (11.4%) and fever (pyrexia; 10.9%). Thirty-five (18.1%) people reported a Hemlibra-related AE, with ISRs being the most common (9.8%).