After approval by the European Medicines Agency (EMA), doctors can use the drug in adults who do not have antibodies against factor VIII or adeno-associated virus serotype 5 (AAV5). Treatment consists of a single administration of valoctocogen roxaparvovec. Long-term efficacy data are lacking, but the effect seems to last at least a few years. This is an advantage over current therapy options, which consist of weekly or monthly injections of factor VIII.
Decrease bleeding risk
Studies have shown that two years after treatment with valoctocogen roxaparvovec, the factor VIII activity is still increased in the majority of patients. Also, the bleeding risk was reduced by 85% and most people no longer needed factor VIII treatment. The study group consisted of 134 adult men without antibodies to factor VIII and without adenoassociated virus serotype 5 (AAV5).
Commonly reported side effects were headache, joint pain and nausea. In addition, elevation of the alanine aminotransferase (ALT) value occurred due to an immune response to the capsid of the AAV5.
Hemophilia is a rare clotting disorder that is caused by a deficiency of clotting factor, in haemophilia A that is factor VIII. This poses a high risk of bleeding. Valoctocogen roxaparvovec consists of the AAV5 virus containing the factor VIII gene. The virus ensures the transfer of the gene in the liver cells, so that these cells can make factor VIII and eliminate the deficiency.