Sanofi and Sobi’s once-weekly haemophilia A treatment approved by FDA

Sanofi and Sobi’s once-weekly haemophilia A treatment, Altuviiio, has been approved by the US Food and Drug Administration (FDA) for the prevention and on-demand treatment to control bleeding episodes, as well as for surgery management for adults and children with the disease.

People living with haemophilia A lack sufficient functioning factor VIII protein to help their blood clot, leading to external bleeding, bruising and bleeding into joints.

Current factor VIII therapies cause protein levels to increase but fall quickly, meaning patients usually have to go in for routine treatment every two days.

As Altuviiio has a half-life that is three to four times longer than standard and extended half-life factor VIII products, it allows for weekly dosing.

Sanofi’s chief executive officer, Paul Hudson, said: "This approval of Altuviiio allows patients and physicians to reimagine living with haemophilia… For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients.”

The FDA’s decision, which marks the first regulatory approval of Altuviiio, was based on results from the phase 3 XTEND-1 study in which the therapy demonstrated superiority to prior factor prophylaxis treatment.

Once-weekly Altuviiio prophylaxis met the study’s primary endpoint, providing significant bleed protection for people with severe haemophilia A with a mean annualised bleeding rate (ABR) of 0.7 and a median ABR of zero.

The treatment also met the key secondary endpoint of the study, with a significant reduction of 77% in ABR versus prior factor prophylaxis based on an intra-patient comparison.

Additional data showed prevention of joint bleeds, and treatment with Altuviiio provided 100% resolution of joints that have recurrent bleeds, such as the knee, ankle or elbow.

Interim data from XTEND-Kids also showed that children aged under 12 years receiving once-weekly Altuviiio for 26 weeks experienced a mean ABR of 0.5 and a median ABR of zero.

Lynn Malec, medical director of Comprehensive Center for Bleeding Disorders and associate investigator at The Versiti Blood Research Institute, said: “This approval marks an important clinical advancement for the haemophilia community because we have an option that can achieve higher levels of factor activity with a single simplified weekly dose.”