HPLC Method for Analysis of Eteplirsen on Primesep B Column

Chromatogram

Description

· High Performance Liquid Chromatography (HPLC) Method for Analysis of Eteplirsen .

Eteplirsen (brand name Exondys 51 ) is an antisense oligonucleotide with the chemical formula C 364 H 569 N 177 O 122 P 30 . It is used for the treatment of some types of Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration and weakness. Eteplirsen specifically targets patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. It works by binding to exon 51 of dystrophin pre-mRNA. This results in the exclusion of exon 51 during mRNA processing, producing a truncated but still functional form of dystrophin protein. Eteplirsen was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in 2016. This approval was based on an increase in dystrophin protein in skeletal muscle observed in some patients treated with Eteplirsen. The FDA has required the manufacturer to conduct a clinical trial to confirm the drug’s clinical benefit. Common side effects include balance disorder and vomiting. The clinical benefit of Eteplirsen, including improved motor function, has not been definitively established.

Eteplirsen can be retained, and analyzed on a Primesep B mixed-mode stationary phase column using an isocratic analytical method with a simple mobile phase of water, Acetonitrile (MeCN), and a sulfuric acid as a buffer. This analysis method can be detected using UV at 260 nm.

Method Parameters

HPLC Column Used