FDA Approves Updated Label for Altuviiio to include Full Data on Pediatric Patients With Hemophilia A


Antihemophilic factor (recombinant) Fc-VWF-XTEN fusion protein-ehtl was initally approved in February 2023 for adults and children with hemophilia A for prophylaxis and on-demand treatment to control bleeding.

The FDA has approved an updated label for antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl (Altuviiio; Sanofi), to include the full results from the phase 3 XTEND-Kids (NCT04759131) study, demonstrating that once-weekly dosing of the drug provides bleed protection in pediatric patients with hemophilia A.1

red blood cells circulating in the blood vessels | Image Credit: Design Cells - stock.adobe.com

Image Credit: Design Cells - stock.adobe.com

The drug was first approved in February 2023 for both children and adults for routine prophylaxis and on-demand treatment as well as perioperative management, according to a press release. The update includes the full results from the study, which highlights the clinical evidence of the drug as a treatment option for all patients.1

“Hemophilia is a lifelong condition, so starting children on an early prophylactic regimen can help them stay ahead of bleeds and reduce other potential complications, such as joint damage and pain. Delivering significant bleed protection with a reduced treatment burden can allow families to spend more time on activities and less time focused on infusion schedules," Mindy Simpson, MD, pediatric hematologist/oncologist and assistant professor of pediatrics at Rush University Medical Center, said in the press release.1

The XTEND-Kids study was an open-label, non-randomized interventional study to evaluate the safety, efficacy, and pharmacokinetics of the drug for patients aged younger than 12 years with severe hemophilia A.1

The drug is a von Willebrand Factor independent recombinant factor VIII therapy for the treatment of hemophilia A and was previously called efanesoctocog alfa. It is the first and only therapy for the condition that provides normal to near normal activity level of over 40% for most of the week with a once-weekly dosing schedule.2

About The Study

Trial Name: Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

ClinicalTrials.gov ID: NCT04759131

Sponsor: Bioverativ, a Sanofi company

Completion Date: January 2023

In September 2023, the FDA accepted a supplemental biologics license application for the drug, showing that the once weekly 50 IU/kg dose of the drug provides effective bleed protection and can be used for pediatric patients across clinical scenarios. It includes 74 patients who received the medication as a prophylaxis for 52 weeks, meeting the primary endpoint of no factor VIII inhibitor development detected and the secondary endpoints of median and mean annualized bleeding rates of 0.00 and 0.6, respectively. The trial also met the maintenance factor levels of being normal or near normal range for approximately 3 days.1,3

The safety profiled showed to be consistent with that in both adults and children, with the most common treatment-emergent adverse events including SARS-CoV-2 positive tests, upper respiratory tract infection and fever. Further, no AEs lead to treatment discontinuation.3 There were no serious allergic reactions, anaphylaxis, or embolic or thrombotic events reported, according to the press release.1

Previously, the drug was granted priority review in September 2022 and was granted breakthrough designation in June 2022.4,5

  1. FDA approves updated Altuviiio label with expanded pediatric data confirming highly effective bleed protection in children with hemophilia A. News release. Sanofi. May 10, 2024. Accessed May 13, 2024. https://www.news.sanofi.us/2024-05-10-FDA-approves-updated-ALTUVIIIO-R-label-with-expanded-pediatric-data-confirming-highly-effective-bleed-protection-in-children-with-hemophilia-A
  2. Pharmacy Times. FDA Approves First-in-Class Factor VIII Therapy for Hemophilia A. February 24, 2023. Accessed May 13, 2024. https://www.pharmacytimes.com/view/fda-approves-first-in-class-factor-viii-therapy-for-hemophilia-a
  3. Gallagher A. FDA Accepts Supplemental Biologics License Application of Altuviiio for Children With Severe Hemophilia A. September 19, 2023. Accessed May 13, 2024. https://www.pharmacytimes.com/view/fda-accepts-supplemental-biologics-license-application-of-altuviiio-for-children-with-severe-hemophilia-a
  4. Gallagher A. FDA Grants Priority Review to Efanesoctocog Alfa for Individuals With Hemophilia A. Pharmacy Times. September 7, 2022. Accessed May 13, 2024. https://www.pharmacytimes.com/view/fda-grants-priority-review-to-efanesoctocog-alfa-for-individuals-with-hemophilia-a
  5. Gallagher A. FDA Grants Breakthrough Therapy Designation to BIVV001 for Hemophilia A Treatment. Pharmacy Times. June 6, 2022. Accessed May 13, 2024. https://www.pharmacytimes.com/view/fda-grants-breakthrough-therapy-designation-to-bivv001-for-hemophilia-a-treatment
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