Sanofi and Sobi’s once-weekly haemophilia A treatment has demonstrated highly effective bleed protection in children with severe forms of the disease, according to late-stage data presented at the International Society on Thrombosis and Haemostasis (ISTH) annual meeting.

The phase 3 XTEND-Kids study evaluated a once-weekly 50IU/kg dose of the factor VIII replacement therapy, Altuviiio, for 52 weeks in previously treated patients aged under 12 years.

The oral presentation at ISTH detailed results from the study and confirmed that the therapy met the primary endpoint, with no inhibitor development to factor VIII detected.

Key secondary endpoints were also met, including annualised bleeding rate and maintenance of factor VIII activity above pre-specified levels.

Sanofi added that the therapy was well-tolerated and demonstrated a safety profile similar to the phase 3 XTEND-1 trial, confirming safety and efficacy in both adults and children.

Haemophilia A is a rare, lifelong bleeding disorder in which a lack of functioning factor VIII protein impairs the body’s blood clotting ability, leading to excessive and spontaneous bleeding into joints that can result in joint damage and chronic pain.

Current factor VIII therapies cause protein levels to increase but fall quickly, meaning patients usually have to go in for routine treatment multiple times a week.

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.

The therapy was approved by the US Food and Drug Administration earlier this year for the prevention and on-demand treatment to control bleeding episodes, as well as for surgery management, in adults and children with haemophilia A.

Karin Knobe, therapeutic area head, rare diseases and rare blood disorders, Sanofi, 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 and reinforce our commitment to developing new treatment options designed to redefine the standard of care for people living with rare blood disorders.”

Reference：
https://www.pmlive.com/pharma_news/sanofi_and_sobis_once-weekly_haemophilia_a_treatment_shows_effective_bleed_protection_in_children_1493852