First Hemophilia Gene Therapy Approved in EU

On August 24, BioMarin Pharmaceutical(BioMarin) announced that the European Commission (EC) has approved the conditionalmarketing of its hemophilia A gene therapy ROCTAVIAN™ (Valoctocogene Roxaparvovec) for the treatment of patients with nohistory of factor FVIII inhibitors and negative for AAV5 antibodies. Adultpatients with severe hemophilia A. This is the world's first approved genetherapy for hemophilia.

Hemophilia A, also known as factor VIIIdeficiency or classic hemophilia, is an X-linked genetic disorder caused by adeletion or defect in the blood clotting protein factor VIII. The main clinicalmanifestations of hemophilia A are repeated bleeding and related complications,of which about 80% are joint hemorrhage. The current standard of care forsevere hemophilia A is long-term, lifelong intravenous infusion of factor VIII,but frequent infusions are extremely inconvenient for patients.

Valoctocogene Roxaparvovec is an AA5 genetherapy that delivers a functional copy of the gene encoding factor VIII intopatients through AAV5, thereby helping patients restore their own ability toproduce factor VIII, reducing the need for ongoing preventive treatment, andpotentially enabling patients to achieve a one-time effect of sex therapy.

Valoctocogene RoxaparvovecClinical Trial Results

The approval is based on overall data fromthe drug's clinical development program, including results from the globalPhase III clinical trial GENEr8-1 (registration number: NCT03370913). Resultsof the GENEr8-1 clinical study showed that the annual bleeding rate (ABR) ofsubjects was significantly reduced after a single infusion of ValoctocogeneRoxaparvovec compared with data from the year prior to enrollment, and the useof recombinant factor VIII (FVIII) protein preparations The frequency decreasedand the FVIII activity in the blood of the patients increased significantly.After 4 weeks of treatment, subjects' annual FVIII use and ABR requiringtreatment were reduced by 99% and 84%, respectively, a statisticallysignificant difference (p<0.001).

In terms of safety, the drug has a goodsafety profile in the trial, with no FVIII factor inhibition, malignancy orthrombosis side effects, and no treatment-related serious adverse events (SAEs)reported.