Roche Announces Analysis of Hemlibra’s Phase III HAVEN 2 Study at ASH meeting

By Rajaneesh K. Gopinath, Ph.D.

After successfully tested by four pivotal phase III HAVEN studies, the bispecific antibody was approved by the FDA in October 2018 for the treatment of Hemophilia A. At the ASH meeting held this month, the primary analyses of HAVEN 2 data were presented.

Hemophilia A (HA) is predominantly an inherited bleeding disorder characterized by a genetic deficiency in an essential clotting factor VIII (fVIII). It is caused by a mutated F8 gene present in the X chromosome and is, therefore, more commonly found in males than females. In healthy individuals, bleeding is controlled by the activation of a coagulation cascade, wherein fVIII brings together other factors, IXa and X to perform blood clotting. Due to low levels of these factors, in the case of HA patients, uncontrolled bleeding occurs either spontaneously or after injuries. The standard treatment that exists for this condition is the replacement of fVIII with recombinant fVIII products. However, patients often develop inhibitory IgG antibodies called fVIII inhibitors that hamper therapy.

Hemlibra (Emicizumab) is a bispecific factor IXa- and X-directed antibody designed to perform the function of fVIII and restore the blood clotting process in HA patients. Created by Chugai Pharmaceutical Co., Ltd., it is co-developed globally by Chugai, Roche, and Genentech. Hemlibra was studied in one of the largest pivotal clinical trials involving HA patients. That includes four phase III HAVEN studies, HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4. Following the positive results from these studies, where Hemlibra significantly reduced bleeds, it received the FDA approval for the treatment of HA with and without fVIII Inhibitors on November 2017 and October 2018 respectively. This makes it the only FDA-approved treatment for this condition that could be self-administered with several dosing options.

At the 60^th American Society of Hematology (ASH) annual meeting held at San Diego from December 1-4, the data from the primary analysis of the HAVEN 2 study was delivered as an oral presentation titled “Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study”. This study evaluated Hemlibra prophylaxis in children younger than 12 years of age with HA with fVIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules. Nearly 77% of children receiving once-weekly dosing experienced zero treated bleeds. This treatment reduced treated bleeds by 99% as compared to prior bypassing agents. Every two weeks and every four weeks dosing also showed significant control of bleeding.

Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development was excited about the developments. “The updated analysis from the HAVEN 2 study supports the potential of Hemlibra to control bleeds at less frequent subcutaneous dosing, providing parents and their children more flexibility to choose a treatment schedule that is right for them,” she said. “Many children with Hemophilia A with factor VIII inhibitors have already experienced the benefits of Hemlibra, and with these new positive data, we are confident that this treatment will continue to make a meaningful difference in their lives.”

On December 4^th, it was also announced that Chugai Pharma Taiwan Ltd., obtained approval from the Taiwan Food and Drug Administration (TFDA) for Hemlibra.

References

1. https://www.roche.com/media/releases/med-cor-2018-12-03b.htm
2. https://ash.confex.com/ash/2018/webprogram/Paper118153.html
3. https://www.drugs.com/history/hemlibra.html
4. https://www.gene.com/assets/frontend/product-information-pages/hemlibra-fact-sheet/Hemlibra-Fact-Sheet_10-02-18.pdf
5. https://www.businesswire.com/news/home/20181203006097/en/Chugais-HEMLIBRA%C2%AE-Approved-Taiwan-Treatment-Hemophilia-Factor
6. https://www.geneonline.com/2018/09/04/emicizumab-promises-weeklybiweekly-prophylaxis-in-hemophilia-a-patients/?doing_wp_cron=1545363254.2336409091949462890625

Author

Rajaneesh K. Gopinath