Arimoclomol citrate is under clinical development by Orphazyme 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 Arimoclomol citrate’s likelihood of approval (LoA) and phase transition for Gaucher Disease Type I took place on 30 Jun 2022, which decreased the likelihood that the drug progresses to the next phase in its clinical pathway and decreased the likelihood of final approval for this indication.

In addition, the same event on 30 Jun 2022 decreased Arimoclomol citrate’s LoA and PTSR for Gaucher Disease Type III.

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

Arimoclomol citrate overview

Arimoclomol citrate (BRX-220) is under development for the treatment of amyotrophic lateral sclerosis (ALS), sporadic inclusion body myositis and Gaucher disease Type 1 and Type 3. The drug candidate is a new molecular entity which is administered through oral route in the form of capsule. It acts by targeting heat shock protein 70.

The drug candidate was also under development for the treatment of Niemann-Pick disease type C, stroke, peripheral neuropathy, insulin resistance, GCase-Parkinson’s disease sporadic inclusion body myositis (s-IBM) and amyotrophic lateral sclerosis.

Orphazyme overview

Orphazyme is a biopharmaceutical company that develops heat shock protein therapies for the treatment of protein-misfolding diseases. The company’s product drug candidate Arimoclomol, is being investigated, for the treatment of neuromuscular disorders such as sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS); and neuropathic lysosomal diseases which includes Niemann-Pick disease Type C (NPC), Gaucher disease and G-Case (glucocerebrosidase) Parkinson’s disease. The company seeks to work in collaboration with academic experts, clinicians and patient organizations to develop its products. The company has operational presence in Chicago, Illinois, the US. Orphazyme is headquartered in Copenhagen, Denmark

Quick View Arimoclomol citrate LOA Data

Report Segments
  • Innovator
Drug Name
  • Arimoclomol citrate
Administration Pathway
  • Oral
Therapeutic Areas
  • Cardiovascular
  • Central Nervous System
  • Genetic Disorders
  • Metabolic Disorders
  • Musculoskeletal Disorders
Key Developers
  • Sponsor Company: Orphazyme
  • Originator: Biorex Research and Development
Highest Development Stage
  • Filing rejected/Withdrawn

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.