Roche has secured approval from the US Food and Drug Administration (FDA) for the use of Hemlibra (emicizumab-kxwh) to treat haemophilia A patients without factor VIII inhibitors.
The indication allows Hemlibra for routine prophylaxis to prevent or decrease the frequency of bleeding episodes in both adults and children.
The drug is a bispecific factor IXa- and factor X-directed antibody formulated for subcutaneous self-administration. It combines the factor proteins to activate coagulation cascade and restore the blood clotting in haemophilia A patients.
The FDA granted breakthrough therapy designation to Hemlibra for the treatment of haemophilia A without factor VIII inhibitors in April this year.
The agency’s latest approval comes after a review of results from the multi-centre, open-label Phase III HAVEN 3 and HAVEN 4 clinical trials performed in 152 and 48 subjects respectively.
Results showed statistically significant and clinically meaningful decrease in treated bleeds with Hemlibra prophylaxis compared to no prophylaxis.
Hemlibra prophylaxis also achieved statistically significant and clinically meaningful reductions across all other bleed-related endpoints in the HAVEN 3 trial, and a clinically meaningful bleeding control in HAVEN 4.
Roche chief medical officer and Global Product Development head Sandra Horning said: “Today’s approval of Hemlibra reflects our commitment to groundbreaking science and the development of medicines with the potential to redefine the standard of care.
“Hemlibra is now the only FDA-approved medicine for people with haemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies.”
Previously, the drug received the FDA approval for prophylactic treatment of people with haemophilia A with factor VIII inhibitors.
It was created by Chugai Pharmaceutical, which is co-developing it with Roche and Genentech.