Altuviiio approved to treat hemophilia A in Japan and Taiwan

Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A.

The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets Altuviiio in the U.S. and most of Asia. The Taiwan Food and Drug Administration approved the therapy in late August.

In children and adults with hemophilia A, the therapy can be used as a once-weekly prophylaxis (preventive) treatment to lower the frequency of bleeds, as an on-demand treatment of bleeding events, and to manage bleeds during surgery.

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“The approval of Altuviiio in Japan and Taiwan represents a major step forward for people living with hemophilia A in those countries,” Brian Foard, an executive vice president at Sanofi, said in a company press release.

Similar uses of Altuviiio were approved by U.S. regulators in February, and an application seeking its approval in the European Union is under review. With approval, Altuviiio in Europe, North Africa, Russia, and most Middle Eastern markets will be sold by Sobi, under a partnership between Sobi and Sanofi.

Hemophilia A is caused by a deficiency of the clotting protein factor VIII (FVIII). Altuviiio is a replacement therapy designed to deliver a working version of FVIII to patients’ blood.

The delivered FVIII has been engineered to bind to others molecules and last longer in the body than the naturally occurring protein, a change that allows the therapy to be given less frequently. As a prophylactic treatment, Altuviiio is administered once weekly via an injection into the bloodstream.

With proper training, the therapy can be administered by patients and caregivers at home.

“The high-sustained factor activity levels will enable patients and physicians to reimagine living with hemophilia,” Foard said. “Altuviiio is a testament to Sanofi’s promise to deliver first-in-class best-in-class therapies that can redefine the treatment paradigm.”

Altuviiio’s approvals largely were based on data from the Phase 3 XTEND-1 clinical trial (NCT04161495), which tested the therapy in 159 people with severe hemophilia A, ages 12 years and older. The results showed that most patients treated once-weekly with Altuviiio had zero bleeds over the year-long study.

They also were supported by findings in the Phase 3 XTEND-Kids trial (NCT04759131), testing Altuviiio as a preventive treatment in 67 boys with severe hemophilia A younger than age 12. Nearly two-thirds of the children were free of bleeds for about a year while using Altuviiio.

The U.S. Food and Drug Administration recently agreed to review a Sanofi request that the therapy’s label be updated to include full results from XTEND-Kids study, which had not finished when U.S. approval was given.