With the addition of Roctavian, gene therapy emerges as another viable treatment alternative. This wide range of options provides patients with a diverse selection to suit their individual circumstances and preferences, but progress is still needed.
“I always say the ultimate unmet need is to cure the disease,” Guy Young, MD, said in an interview with HCPLive. “So something like gene therapy, which is a single infusion, but that will give you a normal level for the rest of your life–we're not probably quite there yet.”
Young, director of the Hemostasis and Thrombosis Center at Children's Hospital Los Angeles, highlighted some of the limitations associated with Roctavian which encompasses approximately 30% of patients with hemophilia A.
“For hemophilia A, we only have 1 subcutaneous drug, which we've mentioned already–Hemlibra,” he said. “It does convert patients to a mild hemophilia phenotype, on the one hand, that's great because they don't bleed very often, but on the other hand, they are still subject to potential bleeds with trauma.”
Patients with hemophilia currently have several treatment choices at their disposal. They can opt for traditional factor concentrates with standard or extended half-life, including Hemlibra, a treatment that has been available for years. They can also explore the new factor concentrate called Altuvio, which boasts an even longer half-life than existing options.
“A subcutaneous drug that could bring the patient's hemostatic status to normal would be would be even better,” Young explained. “So, I think we’re still looking for improved subcutaneous drugs as well, that can really allow patients to live a normal life without living like they have mild haemophilia. We want to do better than that.”