Cerliponase alfa is a Recombinant Enzyme owned by BioMarin Pharmaceutical, and is involved in 8 clinical trials, of which 4 were completed, and 4 are ongoing.

BMN-190 is Tripeptidyl Peptidase 1 (TPP1 ) replacement agent. It restore TPP1 enzyme activity and break down the storage materials that cause CLN2 disease.

The revenue for Cerliponase alfa is expected to reach a total of $3.8bn 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 Cerliponase alfa NPV Report.

Cerliponase alfa is currently owned by BioMarin Pharmaceutical.

Cerliponase alfa Overview

Cerliponase alfa (Brineura, BMN-190) is recombinant human tripeptidyl peptidase 1 (rhTPP1) enzyme. It is formulated as injectable solution for intracerebroventricular (ICV) infusion. Brineura is indicated for the treatment of a specific form of batten disease. Cerliponase alfa is indicated to slow loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. It is based on a patented technology that delivers the treatment directly to the brain. 

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 – Cerliponase alfa

Report Segments
  • Innovator (NME)
Drug Name
  • Cerliponase alfa
Administration Pathway
  • Intracerebral
Therapeutic Areas
  • Central Nervous System
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.