Valoctocogene roxaparvovec is a Gene Therapy owned by BioMarin Pharmaceutical, and is involved in 9 clinical trials, of which 3 were completed, and 6 are ongoing.

Valoctocogene roxaparvovec (BMN-270) expresses the gene which stimulates the production of clotting protein Factor VIII and replace the mutant genes. Thus the drug candidate restores the factor VIII plasma concentrations to levels which is adequate for normal clotting in hemophilia A.

The revenue for Valoctocogene roxaparvovec is expected to reach a total of $21.9bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Valoctocogene roxaparvovec NPV Report.

Valoctocogene roxaparvovec was originated by University College London and St. Jude Children’s Research Hospital and is currently owned by BioMarin Pharmaceutical.

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.

The company reported revenues of (US Dollars) US$1,846.3 million for the fiscal year ended December 2021 (FY2021), a decrease of 0.8% over FY2020. The operating loss of the company was US$82.3 million in FY2021, compared to an operating loss of US$36.8 million in FY2020. The net loss of the company was US$64.1 million in FY2021, compared to a net profit of US$859.1 million in FY2020. The company reported revenues of US$505.3 million for the third quarter ended September 2022, a decrease of 5.3% over the previous quarter.

Quick View – Valoctocogene roxaparvovec

Report Segments
  • Innovator (NME)
Drug Name
  • Valoctocogene roxaparvovec
Administration Pathway
  • Intravenous
  • Intravenous Drip
Therapeutic Areas
  • Hematological Disorders
Key Companies
Highest Development Stage
  • Marketed

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

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.