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Dersimelagon in Erythropoietic Protoporphyrias

In patients with erythropoietic protoporphyria or X-linked protoporphyria, the selective melanocortin 1 receptor agonist dersimelagon significantly increased the duration of symptom-free sunlight exposure, a 102-participant trial shows. “Results from this phase 2 trial support the effectiveness and safety of dersimelagon and its further development as a potential oral treatment option for increasing light tolerance in patients with erythropoietic protoporphyria or X-linked protoporphyria,” conclude the authors.

Patients 18 to 75 years of age with erythropoietic protoporphyria or X-linked protoporphyria were randomly assigned to placebo or dersimelagon 100 or 300 mg once daily for 16 weeks. Based on a primary endpoint of the change from baseline to week 16 in the time to the first prodromal symptom associated with sunlight exposure the study showed: “The mean daily time to the first prodromal symptom associated with sunlight exposure increased significantly with dersimelagon: the least-squares mean difference from placebo in the change from baseline to week 16 was 53.8 minutes in the 100-mg dersimelagon group (P = 0.008) and 62.5 minutes in the 300-mg dersimelagon group (P = 0.003). The results also suggest that quality of life improved in patients receiving dersimelagon as compared with placebo. The most common adverse events that occurred or worsened during treatment were nausea, freckles, headache, and skin hyperpigmentation.”

Source: New England Journal of Medicine