Scenesse increases sunlight tolerance in EPP, XLP patients: Study
Quality of life improvements seen with added light tolerance
Scenesse (afamelanotide) increases the time people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), two types of porphyria, can spend in the sun without pain, which may help improve their quality of life, a study has found.
The study, “Afamelanotide for Treatment of the Protoporphyrias: Impact on Quality of Life and Laboratory Parameters in a US Cohort,” was published as a brief report in Life.
In both EPP and XLP, the body can’t properly produce heme, a molecule that plays a key role in oxygen transport in the body. This leads to the buildup of a chemical called protoporphyrin, largely in the skin.
Light reaching the skin reacts with protoporphyrin, causing itching, a burning sensation, and pain. These symptoms can make life difficult, as patients often have to avoid going outside or being exposed to sunlight or artificial light sources.
Inserted as an implant under the skin, Scenesse, from Clinuvel, is approved in the U.S. and other countries to increase tolerance to the sun and light in adults with a confirmed diagnosis of EPP and a history of adverse skin reactions caused by sunlight.
Simulating melanotropin
Scenesse contains a lab-made hormone that works like the naturally made melanotropin, which stimulates the production of eumelanin in the skin. Eumelanin, a natural dark brown-black pigment, shields against radiation and its reaction with protoporphyrin. This is expected to ease porphyria symptoms.
Researchers at Massachusetts General Hospital in Boston along with colleagues from other U.S. institutions studied the effects of Scenesse in real-world practice in 29 adults — 28 with EPP and one with XLP — by looking at how long it took for sunlight to cause symptoms.
All patients had received at least one dose of Scenesse at the Massachusetts General Hospital Porphyria Center from 2021 to 2022. Over two-thirds (76%) were women, and the median age was 40.
The median number of implants inserted was six. A total of 26 patients received two or more implants during the 20 months of the study. For these patients, the median time they could stay in the sun before symptoms started manifesting increased from 12.5 minutes before treatment to two hours after treatment with Scenesse.
Moreover, all of the patients who received two or more implants “reported a decrease in the frequency and severity of their phototoxic reactions,” or reactions triggered by sunlight exposure, the researchers wrote. However, circulating levels of protoporphyrin remained unchanged.
Consistent with previous observations that Scenesse may improve daily living, patients in this study reported better quality of life after treatment with Scenesse, as assessed by the Patient-Reported Outcomes Measurement Information System 57 (PROMIS-57) and the EPP Quality of Life tools.
Scenesse was also found to be well tolerated, with few side effects aside from nausea, “which is well described,” according to researchers.
The study “demonstrates that [Scenesse] increases the time to phototoxic symptom onset and [quality of life] in patients with protoporphyria.” the researchers wrote. “However, we did not find significant improvements in protoporphyrin levels,” they said.
“Additional investigations into the effect of [Scenesse] on the pathobiology [biological disease mechanisms] of protoporphyria are needed; in particular, to better understand variability in treatment response,” the researchers wrote.
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