Guy Young, MD: Roctavian Approval Expands Options but Eligibility Criteria Remain


While the FDA decision of Roctavian offers patients with hemophilia A more options, the gene therapy eligibility criteria still pose barriers.

The landscape of treatments for hemophilia patients has expanded with the recent approval of Roctavian for hemophilia A, complementing the previous approval for hemophilia B. However, it is important to note that the new gene therapy is only available for adult patients, and therefore, certain limitations apply to eligibility for treatment.

Guy Young, MD, director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, University of Southern California, explained the limitations that come with the treatment, including being under the age of 18.

“If you have an inhibitor, or ever had an inhibitor to factor VIII (FVIII), you will not be able to get (Roctavian) and, at least those who had history of an inhibitor, those were not included as part of the study so it may be challenging to get it for those patients,” he said. “If you have an active inhibitor, you won't be able to get that, in total, that's about 30% of the patients, so it's a sizeable proportion.”

In addition to this, the therapy has been associated with liver complications and those who have antibodies for the vector wouldn't be adequate candidates. However, Young acknowledged it's another therapy to add to patient treatment options.

"Patients have the option of the traditional factor concentrates, standard half-life or extended half-life, they have the option of Hemlibra, which has been around 6 years, and they have the option of a new factor concentrate called Altuvio (Antihemophilic Factor [Recombinant], Fc-VWF-XTEN Fusion Protein-ehtl), which is an even longer half-life than the factors on the market," he said. "And now gene therapy, so they do have quite a lot to choose from."

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