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

Katelyn E Madigan; Sean R Rudnick; Matthew A Agnew; Numra Urooj; Herbert L Bonkovsky

doi:10.3390/ph17010031

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

Pharmaceuticals (Basel). 2023 Dec 25;17(1):31. doi: 10.3390/ph17010031.

Authors

Katelyn E Madigan¹, Sean R Rudnick¹, Matthew A Agnew², Numra Urooj³, Herbert L Bonkovsky¹

Affiliations

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.

Keywords: X-linked protoporphyria; dersimelagon; erythropoietic protoporphyria; oral.

Publication types

Review

Grants and funding

This work was supported in part by funds provided to Atrium Wake Forest Baptist Health from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health. The funding organization had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.