Extremely important for EPP patients
This article discusses SCENESSE® (afamelanotide)’s effect in lowering the risk of liver failure in EPP patients.
In EPP, the high protoporphyrin excretion burden damages the liver cells and the risk increases with higher protoporphyrin concentrations. About 20% of EPP patients show some sign of liver injury and 4% develop life-threatening liver dysfunction.
In vitro and animal studies, afamelanotide improved the synthesis of red blood cell precursors in the bone marrow and the protection of the liver from experimentally induced damage. Dr. Minder’s team from Stadtspital Zürich, Switzerland, analyzed retrospectively the safety laboratory data of 38 EPP Swiss patients on SCENESSE® treatment from 2016 to 2019 and found that the average protoporphyrin concentrations and aspartate aminotransferase (a test for liver function) improved during afamelanotide treatment as compared to before. It was concluded that SCENESSE® (afamelanotide) protects EPP patients from phototoxic reactions and liver injury
Article in Therapeutic Advances in Rare Disease