On 5 September 2022, Clinuvel Pharmaceuticals submitted a marketing authorisation label extension to the European Medicines Agency (EMA) for afamelanotide for the treatment of patients with EPP from age 12 to 17!
The submission is based on four adolescent patients studied in Europe, and the positive outcomes of independent Real-World Evidence studies and the mandatory Post-Marketing Safety and Effectiveness Registry Study, which already led to the positive recommendation for unlimited approval of afamelanotide in November 2019.
Taken together, afamelanotide has been used continuously by adult patients with EPP for more than 16 years, with treatment adherence rates between 94% to 98%, and no major adverse events detected.
Given their limited control over their daily life settings (school schedules etc.) and the social requirements to fit in, young patients are the group with the highest unmet medical need. An expansion of a treatment known to be safe and effective to children from age 12 is a highly welcomed and overdue step to improve the lives of all patients with EPP.
CLINUVEL submits Scenesse® label expansion for adolescents EPP patients. EU regulatory submission follows clinical support for SCENESSE® in EPP patients aged 12-17: Press release
Real-World Evidence studies
Biolcati et al. 2015: Long-term observational study of afamelanotide in 115patients with erythropoietic protoporphyria: https://onlinelibrary.wiley.com/doi/pdf/10.1111/bjd.13598
Wensink et al. 2020: Association of Afamelanotide With Improved Outcomes in Patients With Erythropoietic Protoporphyria in Clinical Practice: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7081144/
Barman-Aksözen et al. 2020: Increased phototoxic burn tolerance time and quality of life in patients with erythropoietic protoporphyria treated with afamelanotide – a three years observational study: https://ojrd.biomedcentral.com/articles/10.1186/s13023-020-01505-6
Unlimited marketing authorisation
European Medicines Agency (2019): SCENESSE Procedural steps taken and scientific information after the authorisation (19 November 2019): Renewal of marketing authorisation; SmPC, Annex II and PL: Based on the review of data on quality, safety and efficacy, the CHMP considered that the benefit-risk balance of SCENESSE in the approved indication remains favourable and therefore recommended the renewal of the marketing authorisation with unlimited validity.