Drug intelligence / Profile preview

ribitol

Development stage
Phase 3
Lead developer
BridgeBio Pharma
Modality
Small Molecules
Administration
Oral
01

Overview

Ribitol is a naturally occurring five-carbon sugar alcohol (pentose alcohol), also known as adonitol. It is formed by the reduction of ribose and occurs in some plants and Gram-positive bacteria as part of teichoic acids. Ribitol is a component of the structure of riboflavin and flavin mononucleotide. As an experimental therapy for certain muscular dystrophies—specifically those related to mutations in fukutin-related protein (FKRP)—ribitol acts as a pre-substrate for glycosyltransferase enzymes involved in the functional glycosylation of α-dystroglycan. Oral administration has been shown to restore matriglycan expression on α-dystroglycan in animal models with FKRP mutations and improve muscle pathology and function dose-dependently[4][6]. Ribitol has received orphan drug designation in Europe for this indication[7].

Other names
adonitolD-adonitolD-ribitolL-ribitol(2R,3s,4S)-pentane-1,2,3,4,5-pentol
02

Targets

FKRP (Fukutin-related protein)α-DG (Alpha-dystroglycan)

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