Drug intelligence / Profile preview

sodium phenylbutyrate + cholecalciferol

Development stage
Unknown
Lead developer
Zevra Therapeutics
Modality
Small Molecules
Administration
Oral, Intravenous, Intramuscular
01

Overview

Sodium phenylbutyrate + cholecalciferol is a combination of two agents, each with distinct mechanisms and clinical uses. Sodium phenylbutyrate is an ammonia scavenger primarily used in the management of urea cycle disorders; it acts by providing an alternative pathway for nitrogen excretion, thereby reducing toxic ammonia levels in the body[4][6][10]. It also functions as a histone deacetylase inhibitor and chemical chaperone, with additional roles in modulating endoplasmic reticulum stress and inflammation[8]. Cholecalciferol (vitamin D3) is a secosteroid hormone precursor that increases intestinal absorption of calcium and phosphate, supporting bone health and immune function[7][9]. The combination has been studied as adjunctive therapy to enhance innate immunity (e.g., upregulation of LL-37 antimicrobial peptide), but there are no approved fixed-dose products or established indications for this specific combination. Both components are available individually as pharmaceuticals or supplements.

Other names
PBA + Vitamin D34-phenylbutyrate + cholecalciferolsodium phenylbutyrate + vitamin D3PBA + cholecalciferol
02

Targets

HDAC (HDAC family)VDR (Vitamin D receptor)

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