Voretigene neparvovec is a Gene Therapy owned by F. Hoffmann-La Roche, and is involved in 7 clinical trials, of which 2 were completed, and 5 are ongoing.

Voretigene neparvovec acts as retinal pigment epithelium 65 (RPE65) activator. Retinal pigment epithelium 65 gene mutation results in loss of cells in the retina over time which leads to total blindness. The therapeutic candidate improves visual and retinal function by delivering the gene for human RPE65 (AAV2-hRPE65v2-1010) into the RPE. It enables the production of the protein that is missing as a result of this genetic mutation.

The revenue for Voretigene neparvovec is expected to reach a total of $3.6bn 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 Voretigene neparvovec NPV Report.

Voretigene neparvovec was originated by University of Iowa Research Foundation and Children’s Hospital of Philadelphia and is currently owned by F. Hoffmann-La Roche. Novartis is the other company associated in development or marketing of Voretigene neparvovec.

Voretigene neparvovec Overview

Voretigene neparvovec or voretigene neparvovec-rzyl (Luxturna) is an adeno-associated virus vector-based gene therapy. It is formulated as injectable suspension, concentrate for solution for subretinal route of administration. Luxturna is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, and also for the treatment of vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations.

Voretigene neparvovec or voretigene neparvovec-rzyl (Luxturna) is under development for the treatment of choroideremia and biallelic RPE65 mutation-associated retinal dystrophy in Japan. It is being developed based on adeno-associated viral (AAV) gene therapy platform.

F. Hoffmann-La Roche Overview

F. Hoffmann-La Roche (Roche) is a biotechnology company that develops drugs and diagnostics to treat major diseases. It provides medicines for the treatment of cancer, other auto-immune diseases, central nervous system disorders, ophthalmological disorders, infectious diseases, and respiratory diseases. The company also offers in vitro diagnostics, tissue-based cancer diagnostics, and diabetes management solutions. Roche conducts research to identify novel methods to prevent, diagnose, and treat diseases. The company offers its products and services to hospitals, commercial laboratories, healthcare professionals, researchers, and pharmacists. Together with its subsidiaries and partners, the company has operations in various countries. Roche is headquartered in Basel, Switzerland.

The company reported revenues of (Swiss Francs) CHF62,801 million for the fiscal year ended December 2021 (FY2021), an increase of 7.7% over FY2020. In FY2021, the company’s operating margin was 28.9%, compared to an operating margin of 31.8% in FY2020. In FY2021, the company recorded a net margin of 22.2%, compared to a net margin of 24.5% in FY2020.

Quick View – Voretigene neparvovec

Report Segments
  • Innovator (NME)
Drug Name
  • Voretigene neparvovec
Administration Pathway
  • Intraocular
Therapeutic Areas
  • Ophthalmology
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.