Policy & Regulation
Genentech Presents a Broad Range of Data for Hemlibra Demonstrating Continued Benefits for People with Hemophilia A at the ISTH 2019 Congress
12 July 2019 - - US-based biotechnology company Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY), has presented new data for Hemlibra (emicizumab-kxwh) across multiple pivotal studies in people with hemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis 2019 Congress on July 6-10 in Melbourne, Australia, the company said.
In total, Genentech presented 21 abstracts from its hemophilia program, including five oral presentations.
Further data from the four pivotal HAVEN clinical trials were presented, demonstrating the long-term safety, efficacy and quality of life benefit of Hemlibra in people with hemophilia A with and without factor VIII inhibitors.
Genentech also presented the first interim analysis from the Phase IIIb STASEY study, reinforcing the safety profile of Hemlibra in adults and adolescents (aged 12 years or older) with hemophilia A with factor VIII inhibitors seen in the HAVEN 1 clinical trial.
Updated data from the pooled HAVEN studies (HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4; n=400), in people with hemophilia A of all ages with and without factor VIII inhibitors, showed that a high proportion of patients experienced zero treated bleeds on Hemlibra, and that this was maintained over a median of 83 weeks.
Across all four HAVEN studies, over 87% of participants had no treated joint bleeds (either spontaneous or due to injury/trauma) and over 92% of participants experienced no spontaneous bleeds in each interval from week 25.
Hemlibra's established safety and tolerability profile was maintained.
Additionally, updated data from the HAVEN 3 and HAVEN 4 studies demonstrate that Hemlibra prophylaxis offers a clinically meaningful improvement in long-term health-related quality of life, versus previous episodic or prophylactic factor VIII treatment, for people with hemophilia A with and without factor VIII inhibitors as measured by the Haem-A-QoL questionnaire.
In the 28 days prior to starting treatment with Hemlibra, 76% and 79% of employed patients from HAVEN 3 and HAVEN 4 studies, respectively, reported no missed days of work.
At week 25 of HAVEN 3 and HAVEN 4, 91 % and 93 % of participants reported no missed workdays, respectively, with these figures remaining stable thereafter.
Interim data from the STASEY study reinforce Hemlibra's safety profile
Results from the first interim analysis of the Phase IIIb STASEY study, including data from 88 patients, reinforce the safety profile of Hemlibra characterized in the pivotal HAVEN 1 study.
HAVEN 1 has formed the basis of Hemlibra's approval in people with hemophilia A with factor VIII inhibitors in over 70 countries worldwide to date. In the STASEY study, in people with hemophilia A with factor VIII inhibitors, no cases of thrombotic microangiopathy or thrombotic events were reported and no new safety signals were identified.
Eighteen (20.5%) patients reported a Hemlibra-related adverse event, of which one was a serious AE (catheter site abscess).
The most common AEs, occurring in 10% or more of people in the STASEY study were injection site reactions (14.8%), joint pain (arthralgia; 13.6%), headache (11.4%) and common cold symptoms (nasopharyngitis; 11.4%).
Bleeding rates in people with hemophilia A with factor VIII inhibitors receiving Hemlibra in the STASEY study were also in line with previously reported observations from the HAVEN 1 study.
Additional factor treatment may not be needed for people on Hemlibra undergoing certain minor surgeries
A retrospective analysis of pooled data across the HAVEN studies suggests that people with hemophilia A with and without factor VIII inhibitors may not need additional preventative (prophylactic) coagulation factor (factor VIII replacement therapy or bypassing agents) when undergoing certain minor surgeries.
The majority of minor surgeries (n=215) were performed without prophylactic coagulation factor (n=141; 65.6 %), and of these, 90.8 % did not result in a treated post-operative bleed.
Of the 18 major surgeries, three were managed without prophylactic coagulation factor, with no post-operative bleeds. The remaining 15 major surgeries were managed with prophylactic coagulation factor, only one of which resulted in a treated post-operative bleed.
Hemlibra is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people with hemophilia A with factor VIII inhibitors in over 70 countries worldwide, based on the results of the pivotal HAVEN 1 and HAVEN 2 studies.
This includes the US in November 2017, EU member states in February 2018 and Japan in March 2018. Hemlibra has also been approved in people with hemophilia A without factor VIII inhibitors in over 40 countries worldwide, based on results from the pivotal HAVEN 3 and HAVEN 4 studies.
This includes the US in October 2018, EU member states in March 2019 and Japan in December 2018. Submissions to other regulatory authorities around the world are ongoing.
The HAVEN clinical trial programme is one of the largest pivotal clinical trial programs in hemophilia A, designed to assess the efficacy and safety of Hemlibra in people with and without factor VIII inhibitors, and its potential to help overcome current clinical challenges: the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access.
HAVEN 1 is a randomised, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of once-weekly subcutaneous administration of Hemlibra prophylaxis compared to no prophylaxis in adults and adolescents (12 years of age and older) with hemophilia A with inhibitors to factor VIII, who were previously treated with bypassing agents on-demand or as prophylaxis.
HAVEN 2 is a multicenter, open-label, clinical study in children younger than 12 years of age with hemophilia A with factor VIII inhibitors.
The study is evaluating the efficacy, safety and pharmacokinetics of once-weekly, every two weeks or every four weeks subcutaneous administration of Hemlibra prophylaxis.
HAVEN 3 is a randomised, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis once weekly or every two weeks versus no prophylaxis (episodic/on-demand factor VIII treatment) in people (12 years of age or older) with hemophilia A without factor VIII inhibitors, who were previously treated with factor VIII therapy either on-demand or as prophylaxis.
HAVEN 4 is a single-arm, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of subcutaneous administration of Hemlibra dosed every four weeks.
The study included adults and adolescents (12 years of age or older) with hemophilia A with or without factor VIII inhibitors who were previously treated with either factor VIII or bypassing agents, on-demand or as prophylaxis.
STASEY is a single-arm, multicenter, open-label, Phase IIIb clinical trial to evaluate the safety and tolerability of Hemlibra prophylaxis in people with hemophilia A with factor VIII inhibitors.
The study included 88 patients (12 years of age or older) who had completed 24 weeks on study or discontinued, receiving subcutaneous Hemlibra 3 mg/kg/week for four weeks, followed by 1.5 mg/kg/week for the remainder of the treatment period.
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients.
It is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly, every two weeks or every four weeks. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech.
Hemlibra is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors.
Hemlibra increases the potential for blood to clot. Patients should carefully follow their healthcare provider's instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment.
Hemlibra may cause the following serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA), including:
Thrombotic microangiopathy. This is a condition involving blood clots and injury to small blood vessels that may cause harm to one's kidneys, brain, and other organs.
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