CLINUVEL has released preliminary data from its CUV052 study showing SCENESSE® is well tolerated in adolescent erythropoietic protoporphyria (EPP) patients, supporting a planned label extension in 2025.
- CUV052 study confirms SCENESSE® safety profile in adolescents matches adults
- Pharmacokinetic data show higher but consistent drug exposure in adolescents
- All eligible adolescent patients opted for continued treatment under special access
- Data to underpin SCENESSE® label extension filing in second half of 2025
- Regulatory engagement ongoing with EMA to expand adolescent indication
Background on SCENESSE® and EPP
CLINUVEL Pharmaceuticals has taken a significant step toward expanding treatment options for adolescent patients suffering from erythropoietic protoporphyria (EPP), a rare inherited genetic disorder characterized by severe phototoxic reactions. SCENESSE® (afamelanotide), already approved for adults, is the first systemic photoprotective drug designed to prevent phototoxicity in EPP patients.
Until now, adolescents aged 12 to 17 have lacked approved access to SCENESSE®, despite the debilitating nature of EPP symptoms. The CUV052 study, initiated in response to European Medicines Agency (EMA) feedback, aimed to evaluate the pharmacokinetics and safety of SCENESSE® in this younger population.
Key Findings from the CUV052 Study
The preliminary results, announced in February 2025, are encouraging. The study enrolled 28 patients evenly split between adults and adolescents, assessing drug tolerability and pharmacological profiles over a 90-day period. SCENESSE® was well tolerated across all participants, with only mild treatment-related adverse events such as headache, nausea, and implant site reactions, all resolving without serious complications.
Pharmacokinetic analyses revealed that adolescents exhibited higher detectable levels of afamelanotide in their bloodstream compared to adults, though these levels remain within historical ranges observed in healthy adult volunteers. This suggests a consistent controlled-release profile of the implant across age groups, supporting the drug's safety and efficacy in younger patients.
Regulatory and Market Implications
With all eligible adolescent patients opting to continue treatment under special access programs, CLINUVEL is poised to submit a label extension application to the EMA in the second half of 2025. This regulatory milestone could open treatment access to approximately 60 known adolescent EPP patients across key European countries including France, Germany, Italy, and the Netherlands.
Dr Dennis Wright, CLINUVEL's Chief Scientific Officer, emphasized the company's commitment to addressing unmet medical needs in this population, noting that the data collected over three years supports a positive risk-benefit profile. The forthcoming full analysis is expected to address regulatory hesitancy and facilitate broader adolescent use.
Looking Ahead
Beyond Europe, CLINUVEL plans to pursue label expansions in other jurisdictions where SCENESSE® is approved, potentially broadening the drug’s market and impact. The company’s ongoing engagement with regulators underscores the strategic importance of this adolescent indication for both patients and shareholders.
As the pharmaceutical landscape increasingly values treatments for rare diseases, CLINUVEL’s progress with SCENESSE® exemplifies how targeted clinical research can unlock new patient populations and commercial opportunities.
Bottom Line?
CLINUVEL’s advancing adolescent data could soon transform SCENESSE® into a standard of care for younger EPP patients.
Questions in the middle?
- Will final CUV052 data confirm the preliminary safety and pharmacokinetic findings?
- How quickly will regulatory agencies approve the label extension following submission?
- What commercial impact will adolescent approval have on CLINUVEL’s revenue trajectory?