Roche Presents Comprehensive Hemlibra Clinical Data Demonstrating Durable Efficacy, Safety, and Quality-of-Life Benefits in Hemophilia A

July 10, 2019/Bio ValleyBIOON/-- Swiss pharmaceutical giant Roche recently announced new data from multiple key studies of its novel hemophilia drug Hemlibra (emicizumab) at the 27th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2019), held in Melbourne, Australia. ThisMeetingRoche presented a total of 21 abstracts from its hemophilia program, including five oral presentations. Data from four pivotal HAVEN clinical studies confirmed the long-term safety, efficacy, and quality-of-life benefits of Hemlibra in patients with hemophilia A, both with and without factor VIII inhibitors. Additionally, Roche announced the first interim analysis of the Phase IIIb STASEY study, which reinforced the safety profile observed in the HAVEN 1 clinical study among adult and adolescent (≥12 years) patients with hemophilia A who have factor VIII inhibitors.

Dr. Sandra Horning, Chief Medical Officer and Global Head of Product Development at Roche, stated: “The data presented at the ISTH Congress continue to reinforce Hemlibra’s potential to redefine the standard of clinical care for patients with hemophilia. The safety data from the first interim analysis of the STASEY study add to the growing body of evidence supporting Hemlibra as an important treatment option for patients with hemophilia.”

Pooled updated data from the HAVEN program (HAVEN 1, HAVEN 2, HAVEN 3, HAVEN 4; n=400) demonstrated that a high proportion of patients with hemophilia A across all age groups, regardless of the presence or absence of factor VIII inhibitors, experienced zero treated bleeds while receiving Hemlibra, with this outcome sustained over a median duration of 83 weeks. Across all four HAVEN studies, more than 87% of patients had no joint bleeds (spontaneous or due to injury/trauma), and more than 92% of patients had no spontaneous bleeds in each evaluation interval starting from Week 25. The established safety and tolerability profile of the drug was maintained.

Furthermore, recent data from the HAVEN 3 and HAVEN 4 studies indicate that, as assessed by the Haem-A-QoL questionnaire, prophylactic treatment with Hemlibra resulted in clinically meaningful improvements in long-term health-related quality of life for patients with hemophilia A, regardless of the presence or absence of factor VIII inhibitors, compared with previous episodic or prophylactic factor VIII therapy. During the 28 days prior to initiating Hemlibra treatment, 76% and 79% of employed patients in the two studies reported no absenteeism; by week 25 of treatment, 91% and 93% of employed patients in the two studies reported no absenteeism, with these figures remaining stable thereafter.

Results from the first interim analysis of this study, which included data from 88 patients, further support the safety profile of Hemlibra observed in the pivotal HAVEN 1 study. To date, HAVEN 1 has served as the regulatory basis for the approval of Hemlibra in more than 70 countries worldwide for patients with hemophilia A with factor VIII inhibitors. In the STASEY study, no cases of thrombotic microangiopathy or thrombotic events were reported among patients with hemophilia A with factor VIII inhibitors, and no new safety signals were identified. Hemlibra-related adverse events (AEs) were reported in 18 patients (20.5%), including one serious AE (catheter-site abscess). The most common AEs occurring in ≥10% of patients in the STASEY study were injection-site reactions (14.8%), arthralgia (13.6%), headache (11.4%), and nasopharyngitis (11.4%). The bleeding rates observed in patients with hemophilia A treated with Hemlibra in the STASEY study were also consistent with previously reported findings from the HAVEN 1 study.

A retrospective analysis of data collected from the HAVEN study program indicated that patients with hemophilia A, regardless of the presence or absence of factor VIII inhibitors, required reduced prophylactic clotting factor (factor VIII replacement therapy or bypassing agents) for certain minor surgical procedures. Most minor surgeries (n=215) were performed without prophylactic clotting factors (n=141; 65.6%), and 90.8% of these did not require postoperative bleeding treatment. Among 18 major surgeries, three were conducted without prophylactic clotting factors, with no postoperative bleeding observed. The remaining 15 major surgeries were managed with prophylactic clotting factors, and only one case required postoperative bleeding treatment.

Hemlibra is a bispecific monoclonal antibody that brings together two proteins essential for activating the natural coagulation cascade and restoring the natural coagulation process—coagulation factors IXa and X—thereby restoring hemostasis in patients with hemophilia A. In clinical studies, Hemlibra has been demonstrated to significantly reduce bleeding events and improve physical function.

To date, Hemlibra has been approved in more than 70 countries and more than 40 countries for use in patients with (based on the HAVEN 1 and HAVEN 2 studies) and without(Based on the HAVEN 3 and HAVEN 4 studies)Hemophilia A patients with factor VIII inhibitors, for the prevention or reduction of the frequency of bleeding episodes.The development of this drug aims to help overcome the current clinical challenges faced by patients with hemophilia A: short duration of action of existing drugs, the development of factor VIII inhibitors, and the need for frequent intravenous infusions.(Bioon.com)

Original Source:Roche Company Website