Vatiquinone is under clinical development by PTC Therapeutics and currently in the Phase III 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 Vatiquinone’s likelihood of approval (LoA) and phase transition for Friedreich Ataxia took place on 14 Oct 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 Vatiquinone Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Vatiquinone overview

Vatiquinone (EPI-743) is under development for the treatment of Alpers-Huttenlocher syndrome, Friedreich ataxia, inherited mitochondrial disorders including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease, mitochondrial diseases and refractory epilepsy. It is administered orally. It is a small molecule that readily crosses into the central nervous system. It works by targeting the enzyme NADPH quinone oxidoreductase 1 (NQO1) and arachidonate 15-lipoxygenase. It was also under development for the treatment of Rett syndrome, Melas syndrome, Leber’s hereditary optic neuropathy and mitochondrial diseases such as Pearson syndrome, a form of mitochondrial myopathy and Tourette syndrome. It was under development for the treatment of cobalamin C deficiency syndrome (methylmalonic acidemia), RARS2 syndrome, Parkinson's disease, metabolic disease (disorders of energy utilization or oxidation-reduction),Leigh's disease in children and noise-induced hearing loss.

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 Vatiquinone LOA Data

Report Segments
  • Innovator
Drug Name
  • Vatiquinone
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Ear Nose Throat Disorders
  • Genetic Disorders
  • Hematological Disorders
  • Metabolic Disorders
  • Musculoskeletal Disorders
  • Ophthalmology
Key Developers
Highest Development Stage
  • Phase III

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.