Introduction
XLP, porphyrias, metabolic disorders
EPP, DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS, PHOTOTOXICITY, METABOLIC DISORDERS, DCSSC, PORPHYRIAS
The porphyrias are a heterogeneous group of rare inherited metabolic disorders caused by enzymatic deficiencies of the heme biosynthetic pathway [Current approaches to the management of EPP and XLP are primarily limited to preventive measures, including sun avoidance and use of protective clothing and sunscreens, posing a substantial burden to patients by requiring significant lifestyle adjustments [Dersimelagon, a novel synthetic orally administered non-peptide small molecule that acts as a selective agonist for MC1R, is currently being investigated for the prevention of phototoxicity in patients with EPP and XLP and inhibition of the fibrotic process in patients with diffuse cutaneous systemic sclerosis (dcSSc). In preclinical studies, dersimelagon exhibited the highest affinity for human MC1R compared with other melanocortin receptors, with a half maximal effective concentration (ECIn a recently completed phase 2 randomized, multicenter, placebo-controlled clinical trial, ENDEAVOR (ClinicalTrials.gov ID: NCT03520036), the safety and efficacy of dersimelagon at doses of 100 mg and 300 mg were investigated [