Eladocagene exuparvovec is under clinical development by PTC Therapeutics and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Eladocagene exuparvovec’s likelihood of approval (LoA) and phase transition for Aromatic l-Amino Acid Decarboxylase (AADC) Deficiency took place on 25 Jul 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Eladocagene exuparvovec Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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.

Quick View Eladocagene exuparvovec LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Eladocagene exuparvovec
Administration Pathway
  • Intracerebral
Therapeutic Areas
  • Genetic Disorders
Key Developers
Highest Development Stage
  • Marketed


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.