Strive for first listing BioMarin suspends haemophilia gene therapy Valrox low-dose study

In order to become the world's first hemophilia gene therapy, BioMarin has announced the abandonment of the development of low-dose Valoccogene roxaparvecThe company now plans to submit accelerated approval applications to the FDA and the European EMA in the fourth quarter of 2019, with the hope of getting it approved by the end of the yearValrox is an AAV gene therapy for adenogenestovirus that can deliver human coagulation factor VIII functional gene to patients to restore the normal secretion of human coagulation factor VIII, thus eliminating or reducing the need for intravenous infusionIn the United States and the European Union, Valrox was granted breakthrough and priority drug qualifications, as well as orphan drug eligibilityIn May, BioMarin announced that Valrox's study of adult patients with severe type A haemophilia met pre-established regulatory review clinical standards in the United States and EuropeIn phase III GENEr8-1 study, after 26 weeks of treatment, 8 patients had a blood clotting factor VIII level of 40 IU/dL above the pre-set levelBut the results also show that Valrox's activity with the human coagulation factor VIII has more than halved from its peak over a three-year follow-up period, from 60 percent to 20 percent of the normal value, so the long-term durability of the treatment has been questionedBioMarin said in a statement that the company chose to suspend the study of Valrox's lower-dose 4e13 vector genome, given that the vast majority of patients were more suited to high-dose treatments for 6e13As for pricing, BioMarin revealed that Valrox's treatment costs about $2 million to $3 million a time and is expected to generate $10 billion to $15 billion a year in revenueBioMarin's second-quarter 2019 financial results, the company said it would continue to publish haemorrhagic control data for the treatment, which is an analysis of phase III clinical interim results from a three-year phase 1/2 clinical study and recently completed haemophiliac type AAccording to BioMarin President of Research and Development Henry Fuchs on an investor conference call, the next trial update for the Valrox treatment program is to complete the recruitment of phase 3 patients by the beginning of the fourth quarter of 2019, which will collect one-year comprehensive treatment data for 130 patients, starting in late 2020 or early 2021currently, the field of hemophilia A gene therapy is on the rise, with BioMarin, Pfizer and Sangamo and Spark among the major players in the fieldFor now, Valrox's main competitor spree may be Pfizer and Sangamo's type A hemophilia gene therapy SB-525Although progress in SB-525 lags slightly, the two companies in July released early data on patients receiving the highest dose of SB-525, which provides human coagulation factor VIII to patients' liver cellsthe two patients who initially gave the highest dose of SB-525 were now followed up at 19 and 24 weeks, respectively, and the activity of the patient's coagulation factor VIII remained at normal levelsAt the same time, the level of coagulation factor VIII also increased in two new patients, one of whom exceeded normal levels after seven weeks of treatment and the other had been relieved of mild haemophiliathe idea that Sangamo gene therapy works quickly, which is a potential advantage that differentiates it from other haemophilia gene therapies In contrast, Phase II data from BioMarin candidates show that factor activity levels in patients can only rise steadily after treatment, reaching normal levels around four months reference source: BioMarin drops lower dose of its hemophilia gene therapy as it eyes submissions by year-end end