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Sanofi’s efanesoctocog alfa may be game-changer for haemophilia A patients

Phase 3 study demonstrated that efanesoctocog alfa met both primary and secondary endpoints.

Research has demonstrated positive safety, efficacy and pharmacokinetic credentials of Sanofi’s efanesoctocog alfa drug.

The therapy is an investigational treatment for haemophilia A and the data showed that it delivered normal to near-normal factor activity levels for most of the week during a once-a-week dosing pattern.

Haemophilia A is a lifelong, rare and genetic condition affecting the ability of a person’s blood to clot, and occurs in about one in 5,000 male births every year while being uncommon in females. Individuals can experience bleeding episodes that can cause irreversible joint damage, severe pain and life-threatening haemorrhages.

Data from the vital XTEND-1 phase 3 study demonstrated that efanesoctocog alfa – also known as ALTUVIIIO – met both primary and secondary endpoints, showing clinically meaningful prevention of bleeds and superior bleed protection compared to prior factor VIII prophylaxis treatment.

Dietmar Berger, global head of development and chief medical officer at Sanofi, reflected: “We are steadfast in our commitment to developing novel treatment options that have a meaningful impact on patients.”

He added: “We are hopeful that efanesoctocog alfa will help deliver on this goal by offering unprecedented factor activity levels with once-weekly dosing, fulfilling its potential as a best-in-class therapy for haemophilia A.”

Associate professor at Michigan Medicine, Angela Weyand, was excited about the potential of the treatment: “Currently, they often need to make trade-offs between bleed protection and dosing frequency.”

“Based on the XTEND-1 study results assessing efanesoctocog alfa, we have the opportunity to provide near normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for haemophilia A,” she concluded.

The treatment is now under priority review by the US Food and Drug Administration and a decision will be made later in the year.

The findings were initially published in The New England Journal of Medicine.