CLINUVEL (ASX: CUV) has released promising preliminary results from its CUV052 study, evaluating SCENESSE® (afamelanotide) in adolescent patients (12–17 years old) with erythropoietic protoporphyria (EPP). The study confirms that the drug is well tolerated in younger patients, with a safety profile consistent with long-term adult use.
Key Findings from the CUV052 Study
- Safety Profile: SCENESSE® was well tolerated in all 28 study participants (14 adults, 14 adolescents), with no serious treatment-related adverse events.
- Biochemical & Pharmacological Data: Drug absorption and release in adolescents were similar to historical data in adults. Drug levels in the blood were slightly higher in adolescents but remained within expected ranges.
- Continued Treatment Demand: All eligible adolescent patients from the study have requested further treatment under special access programs.
Regulatory Pathway for Adolescent Use
SCENESSE® is currently approved for adult EPP patients, but CLINUVEL is working towards expanding its label to include adolescents. Following discussions with the European Medicines Agency (EMA) in 2022 and 2024, the company is compiling additional data from CUV052 to address regulatory concerns.
- Final analysis of the study is expected in H2 2025.
- Regulatory submission for label expansion planned for late 2025.
Expert Commentary
Dr. Dennis Wright, CLINUVEL’s Chief Scientific Officer, emphasized the importance of these results:
“Over the last three years, we have carefully gathered data to support broader access to SCENESSE® for adolescent EPP patients. The preliminary findings confirm that the drug is well tolerated, consistent with adult use. We remain committed to working with regulators to ensure young patients suffering from EPP can access treatment.”
Looking Ahead
With promising safety data and ongoing regulatory engagement, SCENESSE® could soon become available to adolescent EPP patients, providing much-needed relief for those currently deprived of treatment. Final study results in 2025 will be critical in securing approval for this broader patient population.
