FDA asked to update prescribing info on Altuviiio with new trial data

Therapy approved in US earlier this year for all hemophilia A patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) is considering updating the prescribing information for Altuviiio (efanesoctocog alfa) to include final data on a clinical trial that tested the now-approved therapy in children with hemophilia A.

Sanofi, which markets Altuviiio in the U.S., submitted a supplemental biologics license application to the FDA asking for the therapy’s label to be updated. The regulatory agency has now agreed to review that application, with a decision expected May 10, 2024.

“In the U.S., treatment advances over time have given children living with hemophilia the opportunity to move from watching sports as a spectator to becoming an active participant with certain limitations and considerations,” Jeff Schaffnit, head of U.S. Rare Blood Disorders at Sanofi, said in a company press release.

“With Altuviiio, we are building on this momentum by offering a first-of-its-kind option with proven bleed protection and reduced treatment burden for healthcare providers and caregivers of children with hemophilia A,” Schaffnit said.

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No bleeds seen over 1 year on Altuviiio for many Hem A children

Final trial data show therapy effective in children with hemophilia A

Hemophilia A is caused by mutations that cause factor VIII (FVIII), a blood clotting protein, to either be defective or missing altogether in the body. As a result, blood doesn’t clot properly, and patients are more prone to experience prolonged and excessive bleeding.

Altuviiio is a high-sustained replacement therapy that contains a working version of the FVIII protein that can be administered to people with hemophilia A to prevent or control bleeds. The version of FVIII that makes up the therapy contains a series of modifications that increase its stability in the body, allowing for less frequent dosing.

The FDA approved Altuviiio as a treatment for adults and children with hemophilia A earlier this year. That approval was based in large part on data from a Phase 3 trial called XTEND-1 (NCT04161495) that tested Altuviiio in severe hemophilia A patients, ages 12 and older.

The results showed that most patients given once-weekly prophylactic or preventive treatment with Altuviiio had no bleeds over the course of one year.

The FDA’s original approval of Altuviiio also included interim data from another Phase 3 study, called XTEND-Kids (NCT04759131), which tested the therapy in children younger than 12 with severe hemophilia A.

Interim data from XTEND-Kids showed that children given once-weekly prophylactic treatment with Altuviiio for about six months had a mean of 0.5 bleeds per year. Data at the time also suggested Altuviiio’s safety profile in younger patients was similar to that of older patients.

Sanofi’s new application is now asking the FDA to include the final results from this study in the therapy’s prescribing information.

Full data from XTEND-Kids showed that nearly two-thirds of children treated with Altuviiio had no bleeds over the course of a year.

Data also showed that Altuviiio’s safety profile in young children is similar to what had previously been reported in adults and adolescents. Common side effects included headache and joint pain. None of the children in XTEND-Kids developed inhibitors, or neutralizing antibodies against FVIII that can reduce the efficacy of replacement therapies.