Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

Positive Results for Emicizumab in Phase III Studies in Hemophilia A with Inhibitors

By Joanne Van Zuidam | July 14, 2017

Emicizumab decreases incidence of bleeding episodes by 87 percent in patients with Hemophilia A with inhibitors, according to new data from the HAVEN 1 study. Data from HAVEN 1 was released at the International Society of Thrombosis and Hemostasis (ISTH) and published in the New England Journal of Medicine. Interim data from HAVEN 2 study were presented at ISTH as well.

Emicizumab is a bispecific monoclonal antibody designed to bring together factors IXa and X, proteins  which help activate the natural coagulation cascade and restore the blood clotting process, according to Genentech, who is co-developing the treatment with Roche and Chugai Pharmaceuticals.

HAVEN 1 compared emicizumab with on-demand (no prophylaxis) and prophylaxis use of bypassing agents (BPAs) in adults and adolescents with hemophilia A with inhibitors.

The trial included 109 patients 12 years of age or older with hemophilia A with inhibitors to factor VIII, who were previously treated with BPAs on-demand or as prophylaxis.

The primary endpoint was treated bleeds, and results showed a statistically significant and clinically meaningful reduction in bleed rate of 87 percent with emicizumab prophylaxis compared with on-demand treatment with BPAs.

All 12 secondary endpoints were positive, including an intra-patient comparison that showed emicizumab reduced bleed rate by 79 percent compared to prior prophylactic BPAs.

“This is the most significant advancement I have seen during my 20 years working in the field of hemophilia,” said Guy Young, MD, director of the Hemostasis and Thrombosis Program and lead physician for study efforts at Children’s Hospital Los Angeles in a release. Young was part of the international team involved in the HAVEN 1 study. 

Furthermore, a clinically meaningful and statistically significant improvement in health-related quality of life (HRQoL), measured at 25 weeks, using two validated instruments (Haem-A-QoL and EQ-5D-5L), was observed.

Interim results from the single arm HAVEN 2 study in children younger than 12 years of age with hemophilia A with inhibitors who received emicizumab prophylaxis are consistent with the positive results from the HAVEN 1 study.

After a median observation time of 12 weeks, the study showed only one of 19 children receiving emicizumab reported a treated bleed. There were no reported joint or muscle bleeds.

The drug is administered by injection subcutaneously once a week. Current treatment for people with hemophilia A with a severe bleeding phenotype is prophylactic intravenous infusions of factor VIII two to three times weekly.

According to the companies, data from both HAVEN 1 and HAVEN 2 have been submitted for approval consideration to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted Breakthrough Therapy Designation for emicizumab in adults and adolescents with hemophilia A with inhibitors in September 2015. Additional studies evaluating emicizumab in people with hemophilia A both with and without inhibitors are ongoing.


Filed Under: Drug Discovery

 

Related Articles Read More >

Zoliflodacin wins FDA nod for treatment of gonorrhea
FDA approved ENFLONSIA for the prevention of RSV in Infants
First clinical study results of Dupixent for atopic dermatitis in patients with darker skin tones 
Labcorp widens precision oncology toolkit, aims to speed drug-trial enrollment
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE