The Phase III XTEND-Kids clinical trial of once-daily Altuviiio, a recombinant antihaemophilic factor, in children under 12 years of age has met the primary and secondary endpoints.

The detailed data was presented at the Annual Meeting of the International Society on Thrombosis and Haemostasis (ISTH) on 25 June in Montreal, Canada.

Haemophilia A is a rare genetic disorder characterised by low factor VIII levels, a protein required for forming blood clots. In addition, the illness can occur due to an autoantibody response to factor VIII.

According to the US National Organisation for Rare Disorders (NORD), approximately 1 in 5,000 newborn males have haemophilia A, and 60% of these individuals have a severe form of the disorder.

Sanofi therapeutic area head Karin Knobe said: “In an effort to reduce their risk of bleeding episodes, many children living with haemophilia A are currently limited in their ability to fully participate in daily activities.”

“This burden is compounded by the challenge of administering prophylactic treatments intravenously multiple times a week. Today’s XTEND-Kids results reinforce the ability of Altuviiio to provide effective bleed protection with once-weekly dosing.”

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By GlobalData

A total of 74 patients previously treated for severe haemophilia A received once-weekly Altuviiio in the open-label, multicentre Phase III XTEND-Kids (NCT04759131) trial.

No factor VIII inhibitors were detected at 52 weeks, and the mean annualised bleeding rate was 0.89, with 64% of the participants reporting no bleeding episodes.

The Phase III study (NCT04161495) results of Altuviiio in participants 12 years or older with haemophilia A were published in January 2023.

The haemophilia market is currently saturated with multiple treatments, including Genentech’s Hemlibra and Bayer’s Kovalty, with drugs having to distinguish themselves to gain a market share. Gene therapy has been identified as a growing market by GlobalData, with a dominance of as much as 37% of the US haemophilia drug market in 2030.

GlobalData is the parent company of Clinical Trials Arena.

As per Sanofi and Sobi’s collaboration agreement for Altuviiio, final development and commercialisation rights are divided by the region, with Sobi covering Europe, North Africa, Russia, and most of the Middle Eastern markets. Meanwhile, Sanofi covers regions not covered by Sobi, including North America.

Sanofi is also evaluating Altuviiio for Von Willebrand disease (vWD) in an open-label Phase I trial (NCT04770935).