Pegvaliase is a Recombinant Enzyme owned by BioMarin Pharmaceutical, and is involved in 21 clinical trials, of which 11 were completed, 8 are ongoing, and 2 are planned.

Pegvaliase acts as replacement for phenylalanine ammonia lyase. It converts phenylalanine to the harmless metabolites, trans-cinnamic acid and trace ammonia. It reduces the level of phenylalanine (Phe). Excessive level of phenylalanine is causative factor for phenylketonuria. Reduction of phenylalanine (Phe) level by the drug candidate leads to reduction in complication associated with phenylketonuria (PKU).

The revenue for Pegvaliase is expected to reach a total of $9.9bn 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 Pegvaliase NPV Report.

Pegvaliase was originated by IBEX Technologies and is currently owned by BioMarin Pharmaceutical.

Pegvaliase Overview

Pegvaliase (Palynziq) is a derivative of L-alpha-amino acid. It is formulated as pegylated injectable solution for subcutaneous route of administration. Palynziq is indicated for the reduction of phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. Palynziq is indicated to reduce blood phenylalanine concentrations in patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite dietary management.

Pegvaliase is under development for the treatment of hyperphenylalaninemia due to phenylketonuria (PKU).

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 – Pegvaliase

Report Segments
  • Innovator (NME)
Drug Name
  • Pegvaliase
Administration Pathway
  • Subcutaneous
Therapeutic Areas
  • Metabolic 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.