Genentech has announced positive results from a phase 3 trial, showing that Hemlibra (emicizumab-kxwh) continued to demonstrate ‘clinically meaningful’ bleed control in patients with moderate or mild haemophilia A, without factor VIII inhibitors.
The data was presented on 11 July at the 30th international Society on Thrombosis and Haemostasis (ISTH) Annual Congress, and is planned to support a submission to the European Medicines Agency (EMA) to update the label for Hemlibra to include non-severe haemophilia A patients.
The primary analysis from the HAVEN 6 phase 3 study showed that Hemlibra maintained low treated bleed rates across the study period, with 66.7% of participants experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment and 88.9% experiencing no joining bleeds that required treatment.
The study also showed that Hemlibra’s safety profile was consistent with findings across various subpopulations of people with haemophilia A, with no new safety signals observed.
Commenting on the positive results, Levi Garraway, chief medical officer and head of global product development, Genentech, said: “We are proud that Hemlibra continues to redefine the standard of care for more people living with haemophilia A.
“The data presented at ISTH this year underscores Genentech’s commitment to addressing gaps in care for haemophilia A, thereby ensuring that broader populations can potentially benefit from Hemlibra.”
As well as results from HAVEN 6, the company also presented data from CHESS 2 (Cost of Haemophilia across Europe: a Socioeconomic Survey-II) and CHESS PAEDS studies, showing most adults with moderate or mild haemophilia A and more than half of children with Haemophilia A may not receive preventative treatments.
Haemophilia A is a rare, genetic condition that affects the blood’s ability to clot. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
Hemlibra 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 haemophilia A patients.
The preventative treatment is approved to treat people with haemophilia A with factor VIII inhibitors in more than 110 countries and for people without factor VIII inhibitors in more than 95 countries, including the US and Japan for all severities of haemophilia A, and the EU for severe haemophilia A.