Evicenter
P&T meetings

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 description

Clinical presentation - signs and symptoms

9 evidence topics · 12 sources

Cutaneous symptoms: prodromal tingling, burning, and phototoxic pain

“Patients typically present in childhood with prodromal symptoms, including tingling, itching, and burning on sun exposure, which is followed by severe, prolonged phototoxic pain and swelling unless exposure is interrupted. Symptom onset is typically within 10–30 minutes of exposure to visible light.” (opens the source at this quote in a new tab)

“the most commonly reported EPP symptoms after sun exposure were cutaneous burning (289, 89.8%), swelling (273, 84.8%), and tingling (252, 78.3%), and 176 (54.7%) reported a time-to-first symptom of <30 minutes” (opens the source at this quote in a new tab)

“Phototoxic reactions were characterized by pain (97%; 34 patients), burning (97%; 34), tingling (97%; 34), pruritus (83%; 29), allodynia (89%; 31)” (opens the source at this quote in a new tab)

“Prodromal (early warning) symptoms after sunlight exposure, reported by > 50% of participants, included tingling, warmth, itching, sensitivity to touch, and burning; symptoms occurred in ≤ 10 min in 48.8% of adults and 15.2% of adolescents.” (opens the source at this quote in a new tab)

“67.1% of adults and 87.9% of adolescents experienced ≥ 1 phototoxic reaction in the previous 12 months” (opens the source at this quote in a new tab)

Cutaneous symptoms: escalation with continued exposure and the priming phenomenon

“Early symptoms often manifest within a few minutes of light exposure as tingling, itching or burning, which intensify with prolonged light exposure.” (opens the source at this quote in a new tab)

“Repeated light exposure can increase light sensitivity, the so‐called ‘priming phenomenon’.” (opens the source at this quote in a new tab)

Cutaneous symptoms: delayed and often absent visible skin change

“Minimal skin changes or the absence of visible skin changes during reactions to light, including lack of erythema, do not exclude an EPP diagnosis nor suggest low EPP disease burden.” (opens the source at this quote in a new tab)

Cutaneous symptoms: chronic changes of sun-exposed skin

“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.” (opens the source at this quote in a new tab)

“Blistering was reported in 26% of individuals in one large series” (opens the source at this quote in a new tab)

Hepatobiliary symptoms: aminotransferase elevation and gallstones

“Previous studies in cohorts from the United States and Europe have shown elevated serum aminotransferases in 25%–27% of patients with EPP and XLP, and an increased prevalence of cholelithiasis.” (opens the source at this quote in a new tab)

“At the time of enrollment, 49 patients (15.2%) had a history of cholecystectomy” (opens the source at this quote in a new tab)

“Gallstones were commonly reported in patients with EPP or XLP (13.0%).” (opens the source at this quote in a new tab)

“In one series, approximately 22% of individuals with EPP had a known history of gallstones” (opens the source at this quote in a new tab)

“Approximately 20% of patients with EPP develop gallstones, with up to 5% of patients progressing to liver failure” (opens the source at this quote in a new tab)

Hepatobiliary symptoms: liver enzyme, steatosis, and fibrosis findings in a Dutch cohort

“Elevated liver enzymes were found in 6.2% of the patients. Liver steatosis was detected in 29.0%, and significant fibrosis as assessed with liver stiffness measurements was present in 9.6% of patients.” (opens the source at this quote in a new tab)

Hepatobiliary symptoms: higher frequency of abnormal liver enzymes in X-linked protoporphyria

“A natural history study in the US showed that 40% of males with XLP had a history of abnormal liver enzymes compared to 33% of persons with EPP-AR.” (opens the source at this quote in a new tab)

“Recent natural history studies showed that 37.5% of males with XLP and 22.2% of females with XLP had abnormal serum aminotransferases compared to 13.7% of individuals with EPP” (opens the source at this quote in a new tab)

Hematologic symptoms: microcytic anemia and iron deficiency

“A mild hypochromic, microcytic anemia is present in ~30–60% of patients with protoporphyria.” (opens the source at this quote in a new tab)

“Anemia (107 [47.3%]), history of liver dysfunction (62 [27.4%]), and gallstones (53 [23.5%]) were commonly reported.” (opens the source at this quote in a new tab)

“Anemia was present on self-reported medical history in 145 (45.0%)” (opens the source at this quote in a new tab)

“it is estimated that 20%–60% of EPP patients develop mild microcytic anemia whose pathogenesis is unclear” (opens the source at this quote in a new tab)

“In addition, all microcytic patients showed PPIX mean levels significantly higher than normocytic patients” (opens the source at this quote in a new tab)

“Mild anemia with microcytosis and hypochromia or occasionally reticulocytosis can be seen; however, hemolysis is absent or mild.” (opens the source at this quote in a new tab)

Metabolic and bone symptoms: vitamin D deficiency from lifelong sunlight avoidance

“There is an increased recognition of prevalence of vitamin D deficiency in protoporphyria patients due to lifelong sunlight avoidance.” (opens the source at this quote in a new tab)

“All protoporphyria patients are at risk for vitamin D deficiency as a result of their lifelong sun‐avoiding behaviour.” (opens the source at this quote in a new tab)

“Persons with EPP who avoid sun/light are at risk for vitamin D deficiency” (opens the source at this quote in a new tab)