Drug intelligence / Profile preview

Prodarsan

Development stage
Phase 2
Lead developer
Pharming Group
Modality
Small Molecules
Administration
Oral
01

Overview

**Prodarsan** is an investigational oral liquid fixed-dose product developed by DNage for **Cockayne syndrome**, a rare inherited premature-ageing and neurodegenerative disorder. Public reports describe it as a combination of two small molecules, and drug-development databases identify the components as **mannitol and proline**. Its proposed therapeutic rationale was reduction of DNA-damage accumulation and oxidative stress associated with defective DNA-damage responses in Cockayne syndrome; however, no specific direct molecular drug target has been publicly established. A completed Phase 1/2 crossover study evaluated single and multiple escalating oral doses in pediatric patients and compared mannitol pharmacokinetics with intravenous mannitol. ([fiercebiotech.com](https://www.fiercebiotech.com/biotech/prodarsan%C3%A2%C2%AE-granted-orphan-drug-designation-from-fda))

Brand names
Prodarsan

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