{"id":469,"date":"2022-09-10T14:29:25","date_gmt":"2022-09-10T12:29:25","guid":{"rendered":"https:\/\/porphyria.network\/IPPN\/?p=469"},"modified":"2022-09-10T14:43:36","modified_gmt":"2022-09-10T12:43:36","slug":"treatment-for-children-with-epp-available-in-europe-soon","status":"publish","type":"post","link":"https:\/\/porphyria.network\/IPPN\/2022\/09\/treatment-for-children-with-epp-available-in-europe-soon\/","title":{"rendered":"Exciting News: Will treatment for children with EPP aged 12 to 17 be available in Europe soon?"},"content":{"rendered":"\n<p>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! <\/p>\n\n\n\n<p>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. <\/p>\n\n\n\n<p>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. <\/p>\n\n\n\n<p>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.<\/p>\n\n\n\n<h3>Press release<\/h3>\n\n\n\n<p>CLINUVEL submits Scenesse\u00ae label expansion for adolescents EPP patients. EU regulatory submission follows clinical support for SCENESSE\u00ae in EPP patients aged 12-17: <a href=\"https:\/\/www.clinuvel.com\/clinuvel-for-adolescent-epp-patients-20220905\/?mc_cid=e0f8832ea0&amp;mc_eid=3c7f15f779\" target=\"_blank\" rel=\"noreferrer noopener\">Press release<\/a><\/p>\n\n\n\n<p><\/p>\n\n\n\n<h3>Real-World Evidence studies<\/h3>\n\n\n\n<p>Biolcati et al. 2015: Long-term observational study of afamelanotide in 115patients with erythropoietic protoporphyria: <a href=\"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bjd.13598\">https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bjd.13598<\/a><\/p>\n\n\n\n<p>Wensink et al. 2020: Association of Afamelanotide With Improved Outcomes in Patients With Erythropoietic Protoporphyria in Clinical Practice: <a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC7081144\/\">https:\/\/www.ncbi.nlm.nih.gov\/pmc\/articles\/PMC7081144\/<\/a><\/p>\n\n\n\n<p>Barman-Aks\u00f6zen et al. 2020: Increased phototoxic burn tolerance time and quality of life in patients with erythropoietic protoporphyria treated with afamelanotide \u2013 a three years observational study:&nbsp; <a href=\"https:\/\/ojrd.biomedcentral.com\/articles\/10.1186\/s13023-020-01505-6\">https:\/\/ojrd.biomedcentral.com\/articles\/10.1186\/s13023-020-01505-6<\/a><\/p>\n\n\n\n<p><\/p>\n\n\n\n<h3>Unlimited marketing authorisation<\/h3>\n\n\n\n<p>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.<br><a href=\"https:\/\/www.ema.europa.eu\/en\/documents\/procedural-steps-after\/scenesse-epar-procedural-steps-taken-scientific-information-after-authorisation_en.pdf\">https:\/\/www.ema.europa.eu\/en\/documents\/procedural-steps-after\/scenesse-epar-procedural-steps-taken-scientific-information-after-authorisation_en.pdf<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>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&hellip;<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":[],"categories":[31],"tags":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v19.6.1 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Exciting News: Will treatment for children with EPP aged 12 to 17 be available in Europe soon? - International Porphyria Patient Network<\/title>\n<meta name=\"description\" content=\"Exciting News: Will treatment for children with EPP aged 12 to 17 be available in Europe soon? 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