Genentech Reports Positive New Data from Phase 3 Study of Hemlibra Moderate or Mild Hemophilia A
July 12, 2022
Genentech reported positive results from the primary analysis of the phase 3 HAVEN 6 study, which showed that Hemlibra continued to demonstrate a favorable safety profile and effective bleed control in people with moderate or mild hemophilia A, without factor VIII inhibitors.
The data was presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on July 11, 2022, in London and are expected to support a submission to the European Medicines Agency to update the label for Hemlibra to include non-severe hemophilia A patients.
The data presented at ISTH this year underscore Genentech’s commitment to addressing gaps in care for hemophilia A, thereby ensuring that broader populations can potentially benefit from Hemlibra,” said Levi Garraway, chief medical officer and head of Global Product Development.
Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia affects around 20,000 people in the United States, with hemophilia A being the most common form and approximately 50-60 percent of people living with a severe form of the disorder. People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with hemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility, and long-term joint damage.
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. Hemlibra 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 approved to treat people with hemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 95 countries worldwide, including the U.S. and Japan for all severities of hemophilia A, and the European Union for only severe hemophilia A. It has been studied in one of the largest clinical trial programs in people with hemophilia A with and without factor VIII inhibitors, including eight phase 3 studies.
HAVEN 6 is a phase 3, multicenter, open-label, single-arm study evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of Hemlibra in people with moderate or mild hemophilia A without factor VIII inhibitors. The primary analysis included data from 72 participants (69 men and three women) who warranted prophylaxis; 21 had mild hemophilia A without factor VIII inhibitors and 51 had moderate hemophilia A without factor VIII inhibitors at a median follow-up of 55.6 weeks. At baseline, 37 participants were receiving factor VIII prophylactic treatment and 24 had target joints.
The data show that Hemlibra maintained low treated bleed rates across the study period, with 66.7 percent of participants experiencing no bleeds that required treatment, 81.9 percent experiencing no spontaneous bleeds that required treatment, and 88.9 percent experiencing no joint bleeds that required treatment. Model-based annualized bleed rates (ABR) remained low throughout the evaluation period at 0.9.
The results also show that Hemlibra’s safety profile was consistent with findings across various subpopulations of people with hemophilia A, from previous HAVEN and STASEY studies, with no new safety signals observed. The most common adverse event (AE) related to treatment occurring in 10 percent or more people in the HAVEN 6 study was local injection site reactions (ISRs) (16.7 percent). Fifteen people (20.8 percent) reported a Hemlibra-related AE, of which the majority were local ISRs. One participant experienced a grade one thromboembolic event unrelated to Hemlibra. There were no deaths or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favorable safety profile.
Author: Rare Daily Staff
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