Afamelanotide may reduce symptoms, phototoxic reactions in erythropoietic protoporphyria

Treatment with afamelanotide was associated with improved clinical outcomes and a good safety profile in patients with erythropoietic protoporphyria, according to a study published in JAMA Dermatology.

“This study demonstrates that afamelanotide treatment for patients with EPP during routine clinical practice was associated with reduced phototoxicity, more than previously estimated during placebo-controlled trials,” Debby Wensink, MD, of the Porphyria Center Rotterdam, Center for Lysosomal and Metabolic Disease, department of internal medicine, Erasmus MC, University Medical Center in Rotterdam, Netherlands, and colleagues wrote.

The single-center prospective post-authorization safety and efficacy cohort study included 117 patients with erythropoietic protoporphyria (EPP) treated with Scenesse (afamelanotide, Clinuvel Pharmaceuticals), with 115 patients continuing treatment. Afamelanotide was administered as a controlled-release 16 mg subcutaneous implant at least 60 days apart with no more than four implants per year. Data were collected between June 2016 and September 2018.

Outcome measures included time spent outside; quality of life; number, severity and duration of phototoxic reactions; usage of protective clothing; and adverse events.

Results showed that time spent outside with treatment increased by 6.1 hours per week (95% CI, 3.62-8.67) compared with baseline. The improvement in mean quality of life score, 14.01% (95% CI, 4.53%-23.50%), was also significant. Even though phototoxic reactions were less painful, decreasing by 0.85 points (95% CI, –1.43 to –0.26), there was no significant difference in reaction number or duration. One hundred four patients (88.9%) experienced minor self-limiting adverse events (88.9%), with the most reported events being nausea, fatigue/malaise, flushing and nausea with headache.

“Patients with EPP become conditioned to avoid direct sunlight owing to the painful attacks, and such conditioned behavior changes slowly over time if symptoms remain,” the researchers wrote. “The finding that afamelanotide treatment was associated with significantly increased time spent outside with an additional 6 hours per week, with less severe pain, and using less protective clothing is of great clinical relevance as is reflected by the high treatment continuation rate.” – by Erin T. Welsh

Disclosures: Wensink reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.