Avalotcagene ontaparvovec is a Gene Therapy owned by Ultragenyx Pharmaceutical, and is involved in 4 clinical trials, of which 2 were completed, and 2 are ongoing.

DTX-301 acts as ornithine transcarbamylase activator. Ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia, which is formed when proteins are broken down in the body. The drug candidate activates the ornithine transcarbamylase and reduced the abnormal levels of ammonia in the blood, compensating for defects in the intrinsic urea cycle.

The revenue for Avalotcagene ontaparvovec is expected to reach a total of $1.1bn 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 Avalotcagene ontaparvovec NPV Report.

Avalotcagene ontaparvovec was originated by Dimension Therapeutics and is currently owned by Ultragenyx Pharmaceutical.

Avalotcagene ontaparvovec Overview

DTX-301 is under development for the treatment of ornithine transcarbamylase deficiency (urea cycle disorder). It is administered through intravenous route as a concentrate for a solution. The therapy is an adeno-associated virus 8 (AAV8) gene therapy developed based on NAV vector technology.

Ultragenyx Pharmaceutical Overview

Ultragenyx Pharmaceutical (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

The company reported revenues of (US Dollars) US$351.4 million for the fiscal year ended December 2021 (FY2021), an increase of 29.7% over FY2020. The operating loss of the company was US$381.7 million in FY2021, compared to an operating loss of US$330.1 million in FY2020. The net loss of the company was US$454 million in FY2021, compared to a net loss of US$186.6 million in FY2020. The company reported revenues of US$90.7 million for the third quarter ended September 2022, an increase of 1.5% over the previous quarter.

Quick View – Avalotcagene ontaparvovec

Report Segments
  • Innovator
Drug Name
  • Avalotcagene ontaparvovec
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Genetic Disorders
Key Companies
Highest Development Stage
  • Phase III

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.