NPJ005

**NPJ005** is an investigational oral fixed-dose tablet formulation of **5-aminolevulinic acid hydrochloride** and **sodium ferrous citrate** being evaluated in male patients with ATR-X syndrome. Each tablet contains 25 mg 5-aminolevulinic acid hydrochloride and 39.2 mg sodium ferrous citrate; the registered Japanese physician-initiated study evaluates 24 weeks of treatment. 5-aminolevulinic acid is a precursor of heme biosynthesis, while sodium ferrous citrate supplies iron required for heme formation. The proposed biological rationale in ATR-X syndrome includes intracellular porphyrin generation and modulation of DNA G-quadruplex structures, alongside support of heme-dependent mitochondrial respiratory-chain function. ([jrct.mhlw.go.jp](https://jrct.mhlw.go.jp/en-latest-detail/jRCT2051220062))

Other names

5-ALA hydrochloride + SFC5-aminolevulinic acid hydrochloride + sodium ferrous citrate