• BioMarin’s Roctavian can significantly reduce the treatment burden on patients with severe hemophilia A
• Key opinion leaders believe that Roctavian’s benefit could decline over time in hemophilia patients
• If Roctavian is approved, BioMarin is projected to have a market share of 30% by 2027

BioMarin recently presented positive one-year results regarding its GENEr8-1 Phase III pivotal trial, in which adults with severe hemophilia A are treated with the gene therapy Roctavian (valoctocogene roxaparvovec). If approved, patients with severe hemophilia A will have a chance at a normal life with no, or at least very few, ongoing medical interventions, albeit having to pay a high list price, says GlobalData, a leading data and analytics company.

James Donagher, MRes, Oncology and Haematology Analyst at GlobalData, comments: “One-year results from the GENEr8-1 trial state that over 90% of the 134 patients experienced an annualized bleeding rate (ABR) of zero or lower than baseline after four weeks of treatment with Roctavian. This positive data combines well with BioMarin’s previous reports that treatment with Roctavian decreased the need for infusions by 99%. Given that patients with severe hemophilia A are required to take prophylactic (preventative) factor infusions up to three times a week, this could significantly diminish the treatment burden on hemophiliacs. However, patient access could be an issue.”

According to GlobalData’s estimates, Roctavian is expected to cost $3m per dose in the US and EU. Furthermore, gene therapies are designed to be a one-time, curative treatment, yet key opinion leaders (KOLs) believe that there is no way to predict which patients will respond well to gene therapies, and that increased factor levels could decline over time. Therefore, the first gene therapy to market will be the one with the most comprehensive, long-term data.

Nonetheless, BioMarin would benefit from the advantages of developing the first marketed gene therapy for this disorder, projected to have a market share of 30% in 2027. According to GlobalData’s estimates, Roctavian’s global sales are expected to reach $1bn by 2027, while Spark’s second-to-market dirloctocogene samoparvovec is only set to reach $324m by 2027.

Donagher concludes: “BioMarin has recently submitted marketing approval applications to both the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). With EU regulatory bodies expected to make an approval decision in the first half of 2022, Roctavian could be the first gene therapy approved for hemophilia A.”