[Pharmaceutical Directive/Annex XII: Afamelanotide (Reassessment after the deadline: Phototoxicity in erythropoietic protoporphyria (EPP))]

The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
Record ID 32018000678
English, German
Original Title: Arzneimittel-Richtlinie/Anlage XII: Afamelanotid (Neubewertung nach Fristablauf: Phototoxizität bei erythropoetischer Protoporphyrie)
Authors' objectives: The Federal Joint Committee [Gemeinsamer Bundesausschuss (G-BA)] has had the legal task of carrying out an (additional) benefit assessment for all newly approved drugs with new active ingredients immediately after market entry (§ 35a SGB V). The result of this assessment is the basis for deciding how much the statutory health insurance pays for a new drug with a new active ingredient. The G-BA was commissioned to carry out the benefit assessment through the Pharmaceuticals Market Reorganisation Act [Gesetz zur Neuordnung des Arzneimittelmarktes (AMNOG)]. In the context of the early benefit assessment of medicinal products containing new active substances, the following rules apply to orphan drugs: According to the legal requirements (§ 35a SGB V), the additional medical benefit of these drugs is already considered to be proven by the approval. The G-BA determines the extent of the additional benefit for orphan drugs that do not exceed a turnover of 50 million Euros in the last twelve calendar months, on the basis of the approval and the studies justifying the approval.
Authors' results and conclusions: Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). The benefit assessment of scenesse is based on the pivotal study CUV039, a multi-centre, national, placebo-controlled, double-blind phase III RCT evaluating the efficacy and safety of scenesse versus placebo in individuals with EPP. Treatment phase lasted up to six months after inclusion with a follow-up visit up to 12 months after inclusion. The study included 94 patients aged 18 to 74, randomized in a 1:1 ratio to the two treatment groups. The primary endpoint was to investigate the duration of direct sunlight exposure (10:00 a.m. to 6:00 p.m.) on days with no pain. Supporting evidence is provided by the CUV-PASS-001/002 study, an ongoing, non-interventional study in individuals eligible for treatment with scenesse, which is based on a disease registry. No deaths occurred during the course of CUV039 or until the data cut in the PASS study. Across all efficacy endpoint parameters, there is a consistent numerical advantage for scenesse albeit statistically significant only for the patient-specific total time of direct sunlight exposure between 10:00 and 18:00 on days without pain (primary endpoint). As differences in observation duration between treatment arms are not considered in combination with a possible unblinding of subjects and a deviation from the intention-to-treat principle, the results for the morbidity endpoints must be assumed to be highly biased. Quality of life, as measured by the DLQI, showed no statistically significant difference between the scenesse and the placebo group. Due to possible unblinding of subjects, there is a high potential for bias. Regarding safety measures, there is a small numerical disadvantage of scenesse compared to placebo, although the differences were not tested statistically. The PASS study provides no relevant results regarding morbidity. Other non-comparative data from the PASS study provide no new safety findings.
Project Status: Completed
Year Published: 2021
Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Germany
MeSH Terms
  • Protoporphyria, Erythropoietic
  • Sunlight
  • Drug Therapy
  • alpha-MSH
  • Dermatologic Agents
  • Afamelanotide
  • Protoporphyria Erythropoietic
  • Phototoxicity in erythropoietic protoporphyria
  • EPP
  • phototoxicity
Organisation Name: The Federal Joint Committee
Contact Address: Gutenbergstr. 13, 10587 Berlin, Germany
Contact Name: Fachberatung Medizin [Department of Medical Consultancy]
Contact Email: Fachberatung-Medizin@g-ba.de
Copyright: https://www.g-ba.de/sys/impressum/
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