Name: Dersimelagon (MT-7117) in Erythropoietic protoporphyria or X-linked protoporphyria: Long-term morbidity
Published: 2026-09-21T21:42:40-04:00

Product information and disease description Place of product in therapy Disease description

Long-term morbidity

5 evidence topics · 5 sources

Multisystem course requiring longitudinal monitoring

“EPP and XLP are multi-systemic disorders presenting with severe, acute phototoxicity leading to a conditioned behavior of sun avoidance, decreased quality of life, and, in rare cases, liver failure.” (opens the source at this quote in a new tab)

Range of long-term complications

“The complications associated with the protoporphyrias are diverse and include protoporphyria‐related liver failure, gallstones, iron deficiency and vitamin D deficiency, with related reduced bone mineral density and fracture risk.” (opens the source at this quote in a new tab)

Reduced bone mineral density and fracture burden

“BMD is low in EPP patients, with 82.7% of the 139 patients having a Z-score below 0 SD at baseline. Low BMD classified as osteopenia was found in 39.5%, and osteoporosis in 15.3%.” (opens the source at this quote in a new tab)
“There were 50 osteoporosis-related fractures in 34.2% of patients.” (opens the source at this quote in a new tab)
“Among individuals aged 50 years and older, the prevalence of osteoporosis in EPP patients was much higher at 33.3% compared with 6.3% to 14.3% in the general European population” (opens the source at this quote in a new tab)

“In a Dutch cohort study, 36% of patients with EPP were diagnosed with osteopenia and 23% with osteoporosis, while the expected prevalence was 15% and 1%, respectively.” (opens the source at this quote in a new tab)

Chronic cutaneous sequelae

“Multiple episodes of acute photosensitivity may lead to chronic changes of sun-exposed skin (lichenification, leathery pseudovesicles, grooving around the lips) and loss of lunulae of the nails. The dorsum of the hands is most notably affected.” (opens the source at this quote in a new tab)

Greater burden among those with liver disease

“The significant burden imposed by EPP appears to be greater among those with self‐reported liver disease.” (opens the source at this quote in a new tab)
“Among a total of 164 adult participants, 25 (15.2%) self‐reported liver disease and 139 did not.” (opens the source at this quote in a new tab)