Scientists at the Icahn School of Medicine have developed a drug to treat rare conditions that make sun exposure painful. Research published New England Journal of Medicine.
For people with a group of rare genetic diseases, exposure to sunlight can cause excruciating pain. In an early clinical trial, scientists tested the drug in 102 patients with one of two conditions: erythropoietic protoporphyria (EPP) and X-linked protoporphyria (X-PP).
Studies estimate that these diseases affect one in every 75,000 to 200,000 white people. Both conditions are caused by certain genetic abnormalities that cause the chemical protoporphyrin to build up in the blood and in the lining of blood vessels. Sunlight activates protoporphyrin, which causes inflammation, cell damage and severe pain in blood vessels. Both disorders usually appear in childhood.
Standard sunscreen doesn’t offer any protection, but zinc oxide sunscreen can help. Traditionally, the main way of coping with the disorder has been to stay indoors and wear covers when outside.
In 2019, the U.S. Food and Drug Administration approved the first EPP treatment for adults: the hormone drug Scenesse, which increases skin pigmentation. The implant dissolves naturally and should be replaced by a doctor every two months.
The new drug, melagon, is taken orally and does not require frequent doctor visits. Increases skin pigmentation like Scenesse.
Patients were randomly assigned to receive either a cemelagon (higher or lower dose) or placebo tablet daily for 16 weeks. The drug increased comfortable sun exposure by an average of one hour. Generally, within 10-30 minutes, discomfort occurs in people.
Some people have developed nausea, freckles, and headaches. The trial was funded by the pharmaceutical company that developed deselagon.