Original Title: Arzneimittel-Richtlinie/Anlage XII: Sutimlimab (Kälteagglutinin-Krankheit)

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: Sutimlimab (Enjaymo®) is approved for the treatment of hemolytic anemia in adult patients with cold agglutinin disease (CAD). The benefit assessment of sutimlimab is based on the pivotal studies CADENZA (BIVV009-04) and CARDINAL (BIVV009-03). The CADENZA study is a randomized, multicenter, controlled Phase-III-study to investigate the safety and efficacy of sutimlimab compared to placebo in adult patients with hemolytic anemia in CAD (N = 42). The CARDINAL study is a single-arm Phase III study to investigate safety and efficacy in a study population that received a blood transfusion within 6 months prior to the start of the study. After completing part A (N=24) at week 26, it was possible to transfer to the extension study part B (N=22), which lasted at least 2 years. During the RCP, there were no deaths in the CADENZA study and three deaths in the CARDINAL study. Regarding morbidity, data on treatment response, fatigue, thromboembolic events and general health status were presented. In summary, there is a statistically significant advantage of sutimlimab over placebo in the FACIT-fatigue-score at the end of the 26-week RCP of the CADENZA study for the study population predominantly without blood transfusion in the recent medical history regarding a high potential for bias (Mean difference of 10.13 (95% CI: [4.97; 15.29]; p < 0.001)). Data on the generic quality of life instrument SF-12 are available for the outcome category "quality of life", but no relevant influence of Sutimlimab can be derived. Almost all patients in the intervention and control arm experienced an adverse event. There were no statistically significant differences between the treatment arms for the outcomes SAEs, serious AEs (CTCAE grade ≥ 3) and treatment discontinuations due to AEs. There were also no statistically significant or relevant differences for the AESI for which the pharmaceutical company submitted p-values. The risk of bias bias of the CADENZA study is unclear, for the CARDINAL study high, due to the single-arm study design.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/912/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-6044/2023-06-15_AM-RL-XII_Sutimlimab_D-897_EN.pdf

Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/912/#nutzenbewertung

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Germany

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/