Dersimelagon significantly increased duration of symptom-free sunlight exposure in erythropoietic protoporphyria: NEJM

The researchers conducted a phase 2 trial to investigate the efficacy of this drug in these patients in which patients aged 18 to 75 were randomly assigned to ratio of 1:1:1. These patients received a placebo or dersimelagon 100 or 300 mg dose once daily for 16 weeks.

The time to the first prodromal symptom related to sunlight exposure was the primary outcome measured in the study using an electronic diary to record the data.

The study results are as follows:

• One hundred two patients, 93 with erythropoietic protoporphyria and 9 with X-linked protoporphyria, underwent randomization; 90% completed the treatment period.
• The mean daily time to the first prodromal symptom increased with dersimelagon.
• The least-squares mean difference from placebo in the change (baseline to week 16) was 53.8 minutes and 62.5 minutes in the 100-mg and 300-mg dersimelagon group.
• The patients receiving dersimelagon had improved their quality of life.
• The adverse events recorded were nausea, freckles, headache, and skin hyperpigmentation.

They wrote, "At both doses, we found that dersimelagon increased the duration of symptom-free sunlight exposure in erythropoietic protoporphyria or X-linked protoporphyria patients.

The study's limitations include a small sample size, a lack of racial and ethnic diversity, and no long-term follow-up.

Mitsubishi Tanabe Pharma funded the study.

Further reading:

Dersimelagon in Erythropoietic Protoporphyrias. N Engl J Med 2023; 388:1376-1385, DOI: 10.1056/NEJMoa2208754