• Consistent safety profile of SCENESSE in adolescent and adult EPP patients
• Biochemical data in adolescents similar to historical range in healthy adult volunteers
• All eligible patients request further treatment under special access

Clinuvel Pharmaceuticals has announced preliminary results from its post-authorization study of SCENESSE (afamelanotide) in adult and adolescent (12-17-year-old) erythropoietic protoporphyria (EPP) patients (CUV052). SCENESSE was well tolerated by all patients enrolled in the study, with the safety profile of the drug being consistent with that reported in long-term adult use. Biochemical analyses showed that the controlled-release profile of the SCENESSE implant in adolescent patients was similar to that observed in earlier studies in adults. Pending final analyses, data from the CUV052 study will form part of the regulatory package to be submitted to support expanding the SCENESSE indication to include treatment of adolescent patients. The first adolescent patients were treated with SCENESSE in 2021, with strong support from expert physicians. Clinuvel committed to the CUV052 study following feedback from the European Medicines Agency (EMA) that pharmacological and safety data in adolescent patients could support the extension of the SCENESSE label to adolescent EPP patients, who are currently deprived of treatment. A total of 28 EPP patients (14 adults and 14 adolescents) were assessed in the CUV052 study. Preliminary biochemical analyses showed consistent readings among both adults and adolescents, with active drug detectable in blood samples being higher in adolescents compared to adults, although consistent with historical data. Clinuvel is facilitating limited special access to SCENESSE treatment for patients enrolled in the CUV052 study, with all eligible enrolled patients electing to receive further treatment. Final analyses of data from CUV052 are expected in the second half of 2025, with a further submission to the EMA planned to seek expansion of the SCENESSE label.

Clinuvel will incorporate data from the CUV052 study and seek label expansions for SCENESSE in jurisdictions where the drug is approved. The company hopes the complete analyses of data from CUV052 will ultimately address regulatory hesitance and allow wider adolescent use of SCENESSE.