{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,20]],"date-time":"2026-06-20T06:49:26Z","timestamp":1781938166089,"version":"3.54.5"},"reference-count":34,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T00:00:00Z","timestamp":1741824000000},"content-version":"vor","delay-in-days":12,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Photoderm Photoimm Photomed"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Afamelanotide 16\u2009mg (SCENESSE) is the first approved treatment for erythropoietic protoporphyria (EPP). EPP is a rare autosomal recessive inherited disorder of the haem biosynthesis pathway, where patients experience severe and debilitating acute phototoxicity. It affects at least one in 140,000 of the European population. A postauthorisation safety study (PASS) and a disease registry were imposed as conditions of the European marketing authorisation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Evaluate the short\u2010 and long\u2010term safety and clinical effectiveness of afamelanotide 16\u2009mg in EPP patients enrolled in the PASS in Germany.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The PASS (EUPAS13004) is an ongoing observational study collecting safety and effectiveness variables from treated and untreated EPP patients in the European EPP Disease Registry. Patients (<jats:italic>n<\/jats:italic>\u2009=\u2009200, none untreated) received afamelanotide according to the summary of product characteristics. Treatment\u2010emergent adverse events were collected as safety variables. Clinical effectiveness was assessed with the EPP\u2010QoL tool and through treatment continuity.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The short\u2010 and long\u2010term safety and benefit\u2013risk profile of afamelanotide under real\u2010world conditions is consistent with the positive safety profile seen in clinical trials. EPP patients reported a significant increase in QoL compared with baseline values (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001) and 91.0% of patients who started treatment continue being treated. The safety profile of afamelanotide in patients over 70\u2009years of age is consistent with the overall patient population.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Afamelanotide treatment was highly effective and associated with a higher QoL in EPP patients. The study shows a positive safety profile of afamelanotide, with the treatment providing an ongoing clinical benefit.<\/jats:p><\/jats:sec>","DOI":"10.1111\/phpp.13012","type":"journal-article","created":{"date-parts":[[2025,3,14]],"date-time":"2025-03-14T04:23:42Z","timestamp":1741926222000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["German Cohort Observational Study to Investigate the Short\u2010 and Long\u2010Term Safety and Clinical Effectiveness of Afamelanotide 16\u2009mg (<scp>SCENESSE<\/scp>) in Patients With Erythropoietic Protoporphyria (<scp>EPP<\/scp>)"],"prefix":"10.1111","volume":"41","author":[{"given":"Bernhard","family":"Homey","sequence":"first","affiliation":[{"name":"Dept. of Dermatology, Medical Faculty, Porphyria Specialist Center, University Hospital D\u00fcsseldorf Heinrich\u2010Heine\u2010University  D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathrin","family":"Schelonke","sequence":"additional","affiliation":[{"name":"Dept. of Dermatology, Medical Faculty, Porphyria Specialist Center, University Hospital D\u00fcsseldorf Heinrich\u2010Heine\u2010University  D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla Marie","family":"Schlegel","sequence":"additional","affiliation":[{"name":"Dept. of Dermatology, Medical Faculty, Porphyria Specialist Center, University Hospital D\u00fcsseldorf Heinrich\u2010Heine\u2010University  D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniela","family":"Bruch\u2010Gerharz","sequence":"additional","affiliation":[{"name":"Dept. of Dermatology, Medical Faculty, Porphyria Specialist Center, University Hospital D\u00fcsseldorf Heinrich\u2010Heine\u2010University  D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karsten","family":"Weller","sequence":"additional","affiliation":[{"name":"Institute for Allergology, Charite, Universit\u00e4tsmedizin, Berlin, Corporate Member of Freie Universit\u00e4t Berlin und Humboldt Universit\u00e4t zu Berlin, Berlin, Germany; FrauenhoferInstitute for Translational Medicine and Pharmacology IMP, Immunology and Allergology, Berlin, Germany University for Medicine  Berlin Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lea","family":"Kiefer","sequence":"additional","affiliation":[{"name":"Institute for Allergology, Charite, Universit\u00e4tsmedizin, Berlin, Corporate Member of Freie Universit\u00e4t Berlin und Humboldt Universit\u00e4t zu Berlin, Berlin, Germany; FrauenhoferInstitute for Translational Medicine and Pharmacology IMP, Immunology and Allergology, Berlin, Germany University for Medicine  Berlin Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ulrich","family":"St\u00f6lzel","sequence":"additional","affiliation":[{"name":"Clinic for Internal Medicine II Porphyria Specialist Center  Chemnitz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Petra","family":"Staubach\u2010Renz","sequence":"additional","affiliation":[{"name":"University Medical Center of Johannes Gutenberg University  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"Wegner","sequence":"additional","affiliation":[{"name":"University Medical Center of Johannes Gutenberg University  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regine","family":"Keller\u2010Melchior","sequence":"additional","affiliation":[{"name":"University Medical Center of Johannes Gutenberg University  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gillian","family":"Walker","sequence":"additional","affiliation":[{"name":"Clinuvel (UK) Ltd  Leatherhead UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malgorzata","family":"Bochno","sequence":"additional","affiliation":[{"name":"Clinuvel (UK) Ltd  Leatherhead UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Bilbao","sequence":"additional","affiliation":[{"name":"Clinuvel (UK) Ltd  Leatherhead UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,3,13]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamadermatol.2017.1557"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750\u20101172\u20104\u201019"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102796.2010.02236.x"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjd.14600"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V93.6.2105.406k28_2105_2110"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jconrel.2020.08.005"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1177\/26330040211065453"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1080\/17512433.2021.1879638"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102133.2006.07472.x"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545\u2010012\u20109544\u20104"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1684\/ejd.2020.3880"},{"key":"e_1_2_13_13_1","volume-title":"Afamelanotide 16 mg (SCENESSE\u00ae) \u2013 Summary of Product Characteristics (SMPC)\u2010EPAR","author":"CLINUVEL","year":"2021"},{"issue":"3","key":"e_1_2_13_14_1","first-page":"519","article-title":"Enzymological Studies of Melanotropins. 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