Sodium phenylbutyrate IR is under clinical development by Acer Therapeutics and currently in the Phase I 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 Sodium phenylbutyrate IR’s likelihood of approval (LoA) and phase transition for Maple Syrup Urine Disease took place on 27 Dec 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

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

Sodium phenylbutyrate IR overview

Sodium phenylbutyrate is a sodium salt of fatty acid, acts as endocrine and metabolic agent. It is formulated as suspension for oral route of administration. Sodium phenylbutyrate is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC),or argininosuccinic acid synthetase (AS).

Sodium phenylbutyrate immediated release (ACER-001) is under development for the treatment of maple syrup urine disease and urea cycle disorders. The drug candidate is administered as powder orally or through G-tube. The drug candidate is an aromatic fatty acid which acts by targeting the branched chain alpha keto acid dehydrogenase kinase (BCKDK). It is an immediate release formulation.

Acer Therapeutics overview

Acer Therapeutics (Acer), is a Pharmaceutical company. It offers commercialize therapies for patients with serious rare diseases. Its pipeline product portfolio include EDSIVO, for the treatment of vascular ehlers-danlos syndrome; ACER-001, a taste-masked, immediate release formulation of sodium phenyl butyrate for urea cycle disorders (UCD’s) and maple syrup urine disease (MSUD); osanetant, a neurokinin 3 receptor antagonist against induced vasomotor symptoms and emetine for infectious diseases. Acer uses T-cell technology platform for the development of patient-specific T-cell therapies. The company operates manufacturing facility, and research and development facilities in the US and Canada. Acer is headquartered in Newton, Massachusetts, the US.

Quick View Sodium phenylbutyrate IR LOA Data

Report Segments
  • Innovator (Non-NME)
Drug Name
  • Sodium phenylbutyrate IR
Administration Pathway
  • Oral
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.