5-year long-term effect observation of BioMarin's hemophilia gene therapy phase III clinical trial, with positive data

BioMarin's therapy, called valoctocogene roxaparvovec or 'valrox', in the Phase 3 GENER8-1 trial, 8 of 20 adults with severe hemophilia A had sufficient improvements in factor VIII levels to meet prespecified regulatory review standards
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In June 2019, BioMarin reported three years of data on its gene therapy for hemophilia A, which was used to support its applications in Europe and the US, however, its long-term therapeutic value has also been called into question
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On January 9, 2022, BioMarin Pharmaceutical announced that valoctocogene roxaparvovec has achieved positive results in an ongoing Phase III clinical trial
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This is an investigational gene therapy using an AAV5 viral vector to deliver a transgene expressing coagulation factor VIII for the treatment of adults with severe hemophilia
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However, it was also seen that the activity of FVIII continued to decrease with prolonged follow-up

However, the gene therapy will have a high price once it comes out, see: BioMarin plans to price its hemophilia A gene therapy Valrox at $2 million to $3 million