Valoctocogene roxaparvovec is under clinical development by BioMarin Pharmaceutical and currently in Pre-Registration for Hemophilia A (Factor VIII Deficiency). According to GlobalData, Pre-Registration drugs for Hemophilia A (Factor VIII Deficiency) have a 93% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Valoctocogene roxaparvovec’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Valoctocogene roxaparvovec overview

Valoctocogene Roxaparvovec (Roctavian) is a gene therapy medicinal product that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). It is formulated as a solution for infusion for intravenous route of administration. It is indicated for the treatment of severe hemophilia A (congenital factor viii deficiency) in adult patients without a history of factor viii inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (aav5).

Valoctocogene roxaparvovec (BMN-270) is under development for treatment of hemophilia A. It is administered as an intravenous infusion. The drug candidate is a recombinant adeno-associated virus (AAV) 5 coding for human-coagulation factor VIII.

BioMarin Pharmaceutical overview

BioMarin Pharmaceutical (BioMarin) develops and commercializes innovative medicines for the treatment of rare genetic diseases and medical conditions such as Duchenne muscular dystrophy (DMD), phenylketonuria (PKU), achondroplasia, late infantile neuronal ceroid lipofuscinosis (CLN2) and hemophilia A. The company focuses on developing breakthrough treatments for debilitating and life-threatening diseases. Its product category consists of multiple clinical and preclinical offerings which include Naglazyme-Mucopolysaccharidosis Type VI (MPS VI), Aldurazyme-MPS I and others. The company operates through its manufacturing facilities in Novato, California. It conducts business operations in the US, Europe, Latin America, the Middle East, and Asia-Pacific. BioMarin is headquartered in San Rafael, California, the US.

For a complete picture of Valoctocogene roxaparvovec’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.