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Sanofi ALTUVIIIO hemophilia A treatment: FDA accepts sBLA based on pediatric phase 3 data

By Brian Buntz | September 12, 2023

Lyon France, 3 October 2017: White logo of the french multinational pharmaceutical company Sanofi on their glass office building in Lyon France

[Keitma/Adobe Stock]

The FDA has accepted Sanofi’s supplemental Biologics License Application (sBLA) for ALTUVIIIO, a novel, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. The FDA based the decision on positive final data from the pivotal Phase 3 XTEND-Kids trial in children under 12 with hemophilia A.

ALTUVIIIO (efanesoctocog alfa) is the first Factor VIII replacement therapy to offer bleed protection with once-weekly dosing. In February, Sanofi announced that FDA had approved the drug for routine prophylaxis and on-demand treatment to control bleeding episodes. Its use also extends to perioperative management (surgery) for adults and children with hemophilia A.

Sanofi developed in collaboration with Sobi. Barclays projects that the drug will top €2 billion in peak sales.

Supportive findings from XTEND-kids study

The current FDA approval is supported by interim data from XTEND-Kids. The sBLA seeks to update ALTUVIIIO’s label with the final pediatric results.

In the XTEND-Kids study, 74 patients on weekly ALTUVIIIO prophylaxis experienced a median annual bleed rate of zero. ALTUVIIIO also maintained factor activity levels above 40% for around three days. The safety profile was similar to adults with no concerning side effects.

Traditionally, hemophilia A is treated with routine prophylaxis, which involves regularly infusing patients with clotting factor concentrates to prevent bleeding episodes. This approach is common in individuals with severe hemophilia A while on-demand treatment provides intermittent support to individuals with mild or moderate hemophilia A who have no prior bleeding history.

For patients with the condition, frequency of standard factor replacement can be a major treatment burden, often requiring injections multiple times per week. This can be especially challenging for children.

Traditional Hemophilia A therapies 

Hemophilia A is a genetic disorder that affects the blood’s ability to clot properly. The main treatment for severe hemophilia A involves replacing the clotting factor you need through a tube in a vein. This replacement therapy can be given to treat a bleeding episode in progress. It can also be given on a regular schedule at home to help prevent bleeding episodes. Some people receive continuous replacement therapy. Replacement clotting factor can be made from donated blood. Similar products, called recombinant clotting factors, are made in a laboratory, not from human blood.

Other therapies involved to treat the condition include desmopressin, which is used for some forms of mild hemophilia. In addition, emicizumab (Hemlibra) is a newer drug that doesn’t include clotting factors that can help prevent bleeding episodes. Other therapies include clot-preserving medications known as anti-fibrinolytics, fibrin sealants and physical therapy.


Filed Under: Biologics, Hematology, Pediatrics, Regulatory affairs
Tagged With: ALTUVIIIO, Biologics License Application, Factor VIII replacement therapy, hemophilia A, Pediatric results, Sanofi, XTEND-Kids trial
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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