Dersimelagon Beneficial for Erythropoietic, X-Linked Protoporphyria

THURSDAY, April 13, 2023 (HealthDay News) -- For patients with erythropoietic protoporphyria or X-linked protoporphyria, dersimelagon at doses of 100 and 300 mg significantly increases the duration of symptom-free sunlight exposure, according to a study published in the April 13 issue of the New England Journal of Medicine.

Manisha Balwani, M.D., from the Icahn School of Medicine at Mount Sinai in New York City, and colleagues conducted a randomized phase 2 trial involving patients with erythropoietic protoporphyria or X-linked protoporphyria aged 18 to 75 years to examine the efficacy and safety of dersimelagon with respect to the time to onset and the severity of symptoms associated with sunlight exposure. A total of 102 patients were randomly assigned to receive placebo or dersimelagon at 100 or 300 mg once daily for 16 weeks in a 1:1:1 ratio; 90 percent completed the treatment period.

The researchers observed a significant increase in the mean daily time to the first prodromal symptom associated with sunlight exposure with dersimelagon: least-squares mean difference from placebo in the change from baseline to week 16, 53.8 and 62.5 minutes in the 100- and 300-mg dersimelagon groups, respectively. Patients receiving dersimelagon had improved quality of life compared with those receiving placebo. Nausea, freckles, headache, and skin hyperpigmentation were the most common adverse events that occurred or worsened during treatment.

"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," the authors write.

The study was funded by Mitsubishi Tanabe Pharma, the manufacturer of dersimelagon.

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