Adrabetadex (VTS-270): A Comprehensive Review of its Development for Niemann-Pick Disease Type C1

I. Introduction to Adrabetadex (VTS-270)

A. Overview of the Compound

Adrabetadex represents an investigational therapeutic agent developed primarily for Niemann-Pick Disease Type C (NPC). It is the United States Adopted Name (USAN) for a specific formulation of (2-hydroxypropyl)-β-cyclodextrin (HPβCD).[1] HPβCD is a chemically modified cyclic oligosaccharide derived from β-cyclodextrin, belonging to the chemical class of beta-cyclodextrins and ethers.[2] Although classified as a small molecule for regulatory and database purposes [4], HPβCD itself is not a single molecular entity but rather a complex mixture of β-cyclodextrin molecules with varying degrees of hydroxypropylation.[3] The specific formulation investigated extensively in clinical trials for NPC was designated VTS-270.[3] Other identifiers associated with this substance include Kleptose HPB.[1] The development and nomenclature history reflect its transition from a chemical entity (HPβCD) to a specific investigational drug product (VTS-270) and finally to a formally adopted name (Adrabetadex).

B. Identification and Properties

Precise identification is crucial for tracking and researching this investigational compound. Key identifiers and basic properties are summarized in Table 1.

Table 1: Adrabetadex Key Identifiers and Properties