Eladocagene exuparvovec is a Gene Therapy owned by PTC Therapeutics, and is involved in 5 clinical trials, of which 3 were completed, and 2 are ongoing.

Eladocagene exuparvovec stimulates the synthesis of aromatic l-amino acid decarboxylase (AADC) enzyme. This enzyme then stimulates the synthesis of the neurotransmitters dopamine and serotonin whose deficiency is causative factor for the disease. These neurotransmitters (dopamine and serotonin) then stimulate the motor and mental developments.

The revenue for Eladocagene exuparvovec is expected to reach a total of $4.2bn 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 Eladocagene exuparvovec NPV Report.

Eladocagene exuparvovec was originated by National Taiwan University and is currently owned by PTC Therapeutics.

Eladocagene exuparvovec Overview

Eladocagene exuparvovec (Upstaza) is a gene therapy product. It is formulated as solution for intracerebral route of administration. Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Eladocagene exuparvovec is under development for treatment of aromatic l-amino acid decarboxylase (AADC) deficiency. The therapeutic candidate is administered by intracerebral (into the putamen of the brain) route. The therapeutic candidate employs Adeno-associated virus serotype 2 (AAV2) to carry the human AADC gene.

PTC Therapeutics Overview

PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren) and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. The company operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.

The company reported revenues of (US Dollars) US$538.6 million for the fiscal year ended December 2021 (FY2021), an increase of 41.4% over FY2020. The operating loss of the company was US$374.4 million in FY2021, compared to an operating loss of US$431.8 million in FY2020. The net loss of the company was US$523.9 million in FY2021, compared to a net loss of US$438.2 million in FY2020. The company reported revenues of US$217.1 million for the third quarter ended September 2022, an increase of 31.2% over the previous quarter.

Quick View – Eladocagene exuparvovec

Report Segments
  • Innovator (NME)
Drug Name
  • Eladocagene exuparvovec
Administration Pathway
  • Intracerebral
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.