Vestronidase alfa is a Recombinant Enzyme owned by Ultragenyx Pharmaceutical, and is involved in 9 clinical trials, of which 5 were completed, and 4 are ongoing.

Vestronidase alfa is an enzyme replacement therapy. It is intended to provide exogenous beta-glucuronidase for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular internalization of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.

The revenue for Vestronidase alfa is expected to reach a total of $665m 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 Vestronidase alfa NPV Report.

Vestronidase alfa was originated by Saint Louis University and is currently owned by Ultragenyx Pharmaceutical.

Vestronidase alfa Overview

Vestronidase alfa-vjbk (MepseVII) is a lysosomal beta-glucuronidase enzyme. It is formulated as solution and concentrate solution for intravenous route of administration. MEPSEVII is indicated for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatrics and adults.

Vestronidase alfa is under development for the treatment of mucopolysaccharidosis VII (MPS VII) (Sly Syndrome) in Japan. The drug candidate is administered as intravenous drip.

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 – Vestronidase alfa

Report Segments
  • Innovator (NME)
Drug Name
  • Vestronidase alfa
Administration Pathway
  • Intravenous
  • Intravenous Drip
Therapeutic Areas
  • Genetic 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.