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Dersimelagon (MT-7117)

Erythropoietic protoporphyria or X-linked protoporphyria

Manufacturer
LEO Pharma
Regulatory submission
NDA submitted June 2026
Last updated
100 sources

Product information and disease descriptionPlace of product in therapyDisease descriptionBurden of Erythropoietic protoporphyria or X-linked protoporphyria

Economic impact of Erythropoietic protoporphyria or X-linked protoporphyria on families

3 evidence topics · 3 sources

Quantified family out-of-pocket costs and caregiver productivity loss

No evidence found.

Family adjustments described by parents of affected children

“All parents described a frustrating diagnostic odyssey before their child was finally diagnosed with EPP. They all agreed they felt relief following the diagnosis of EPP in their children, as well as validation that their child's symptoms were real.” (opens the source at this quote in a new tab)

“Parents had to make adjustments to their family's lives to accommodate for EPP such as driving to places in two separate cars, one with window tints and one without, and planning family activities outdoors in the evening.” (opens the source at this quote in a new tab)

“Most parents thought that their children initially struggled with fitting in socially, specifically at school, as they were physically isolated during activities such as recess, which resulted in social isolation.” (opens the source at this quote in a new tab)

Time lost from work and school in households affected by liver disease

“The mean number of hours missed from work and school in the past month was higher for those with liver disease (work: 10.9 h; school: 7.7 h) than those without liver disease (3.6 h for both).” (opens the source at this quote in a new tab)

Education of the family and school environment as a component of care

“It is crucial and complex for protoporphyria patients and their families to inform and educate their social network, to raise awareness and understanding of the limitations and the necessary support.” (opens the source at this quote in a new tab)

“Supporting the patients and their families in educating them about their social, school and work environments is an important part of care.” (opens the source at this quote in a new tab)