Drug intelligence / Profile preview

sarizotan

Development stage
Phase 3
Lead developer
Newron Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

Sarizotan is a small molecule drug developed as a selective 5-HT1A (serotonin) receptor agonist and D2 (dopamine) receptor antagonist. It was originally investigated for its antipsychotic effects and for reducing dyskinesias associated with long-term levodopa treatment in Parkinson's disease. Sarizotan also demonstrated high affinity for D3 and D4 dopamine receptors. The drug was further explored as an oral therapy to treat breathing abnormalities in Rett syndrome, based on its ability to modulate serotonin and dopamine signaling in the brain. Despite promising preclinical results, clinical development for Parkinson's disease was discontinued after Phase III trials failed to meet efficacy endpoints. Later, Newron Pharmaceuticals pursued development of sarizotan for Rett syndrome, receiving orphan drug designation from the FDA.

Other names
sarizotan [INN]
02

Targets

HTR1A (Serotonin receptor 1A (5-hydroxytryptamine receptor 1A))DRD2 (Dopamine D2 Receptor)DRD3 (Dopamine receptor D3)DRD4 (Dopamine D4 Receptor)KCNH2 (Voltage-gated potassium channel subfamily H member 2)

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