Drug intelligence / Profile preview

ronopterin

Development stage
Phase 3
Lead developer
Vasopharm
Modality
Small Molecules
Administration
Intravenous
01

Overview

Ronopterin is a small molecule neuroprotective drug developed primarily for the treatment of traumatic brain injury (TBI). It acts as a selective inhibitor of inducible nitric oxide synthase (iNOS) by binding to the endogenous tetrahydrobiopterin (BH4) binding site on the enzyme. This inhibition reduces excessive nitric oxide and radical production, decreases extracellular brain glutamate levels, and limits cell damage and inflammatory responses in the injured brain. Ronopterin has orphan drug designation for brain injuries in Europe and has reached phase III clinical trials for TBI. The drug is administered intravenously and has shown promising neuroprotective effects in preclinical models and early-phase clinical studies.

Brand names
Ronopterin
Other names
4-aminotetrahydrobiopterinronopterinaronopterine
02

Targets

NOS3 (eNOS)NOS2 (Nitric Oxide Synthase 2)NOS1 (nNOS)

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