Name: Dersimelagon (MT-7117) in Erythropoietic protoporphyria or X-linked protoporphyria: Management of hepatobiliary complications
Published: 2026-09-21T21:42:40-04:00

Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Management of hepatobiliary complications

3 evidence topics · 2 sources

Course of protoporphyric hepatopathy and monitoring recommendation

“Approximately 1–5% of patients with protoporphyria develop rapid and severe hepatobiliary disease characterized by an acute cholestatic hepatitis with liver failure (often called “EPP hepatic crisis”), or a slowly progressive biliary cirrhosis and end stage liver disease; these more advanced manifestations of liver disease due to protoporphyria are collectively referred to as “protoporphyric hepatopathy” (opens the source at this quote in a new tab)
“These patients almost invariably require liver transplantation” (opens the source at this quote in a new tab)
“Therefore, it is recommended that liver chemistries should be monitored at least yearly in patients with protoporphyria to detect early stages of liver dysfunction starting at the time of diagnosis of protoporphyria ( Figure 4” (opens the source at this quote in a new tab)
“We recommend TPE for patients with advanced protoporphyric hepatopathy to decrease plasma porphyrin levels and thereby reduce liver damage in order to bridge patients to liver transplant and/or hematopoietic stem cell transplant (see below” (opens the source at this quote in a new tab)
“Recurrent protoporphyric hepatopathy in the transplanted liver has been reported in 65 to 80% of patients in case series from the US, Australia, and Europe, with 43% showing evidence of recurrence by one year post-transplant” (opens the source at this quote in a new tab)

Escalating interventions for hepatic decompensation

“As a first measure, any hepatotoxins should be eliminated. Depending on the severity of the liver disease, phlebotomies, exchange transfusions and ultimately liver transplantation with subsequent haematopoietic stem cell transplantation (HSCT) are therapeutic options, whereby multidisciplinary management including porphyria experts is mandatory.” (opens the source at this quote in a new tab)

Genotype-dependent effect of iron supplementation

“In EPP1, even in cases of biochemically determined iron deficiency, supplementation of iron may stimulate PPIX production, resulting in an increase in photosensitivity and the risk of cholestatic liver disease. However, for patients with XLEPP, iron supplementation can reduce PPIX levels, phototoxicity and liver damage.” (opens the source at this quote in a new tab)