Illuminating Dersimelagon: A Novel Agent in the Treatment of Erythropoietic Protoporphyria and X-Linked Protoporphyria

Author:

Madigan Katelyn E.1ORCID,Rudnick Sean R.1,Agnew Matthew A.2,Urooj Numra3,Bonkovsky Herbert L.1ORCID

Affiliation:

1. Section on Gastroenterology & Hepatology, Department of Medicine, Wake Forest University School of Medicine, Winston-Salem, NC 27157, USA

2. Department of Internal Medicine, Wake Forest University School of Medicine, Winston-Salem, NC 27157, USA

3. Department of Medicine, Parkview Health, Fort Wayne, IN 46845, USA

Abstract

Erythropoietic protoporphyria (EPP) is a genetic disorder stemming from reduced ferrochelatase expression, the final enzyme in the pathway of heme biosynthesis. A closely related condition, X-linked protoporphyria (XLP), bears similar clinical features although it arises from the heightened activity of δ-aminolevulinic acid synthase 2 (ALAS2), the first and normally rate-controlling enzyme in heme biosynthesis in developing red blood cells. Both of these abnormalities result in the buildup of protoporphyrin IX, leading to excruciating light sensitivity and, in a minority of cases, potentially fatal liver complications. Traditionally, managing EPP and XLP involved sun avoidance. However, the emergence of innovative therapies, such as dersimelagon, is reshaping the therapeutic landscape for these conditions. In this review, we summarize salient features of the properties of dersimelagon, shedding light on its potential role in advancing our understanding of treatment options for EPP and XLP.

Funder

Atrium Wake Forest Baptist Health from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health

Publisher

MDPI AG

Subject

Drug Discovery,Pharmaceutical Science,Molecular Medicine

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