Original Title: Arzneimittel-Richtlinie/Anlage XII: Lonapegsomatropin (Wachstumsstörung durch Wachstumshormonmangel, ≥ 3 bis < 18 Jahre)

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: Lonapegsomatropin is approved for the treatment of growth failure in children and adolescents aged 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency (GHD)). The benefit assessment and an amendment are based on the heiGHt and CT-301-CN studies. Both are randomised, open phase III studies that investigate the efficacy and safety of lonapegsomatropin compared to somatropin in prepubertal children for a duration of 52 weeks. In total, 316 patients with GHD (age from 3 to 14 years) were randomised in a 2:1 ratio (lonapegsomatropin: somatropin). Zero deaths were reported. Both studies showed a statistically significant change in height standard deviation score (SDS) in favour of lonapegsomatropin compared to somatropin over 52 weeks. Meta-analysis showed a similar result in favour of lonapegsomatropin with an LS-MWD of 0.15 (95% CI: [0.07; 0.23]); p = 0.0002. No QoL data was collected. AEs were reported by 77 % of patients who received lonapegsomatropin and 70 % of patients who received somatropin in the heiGHt study, whereas 98% vs. 94% reported AEs in the CT-301-CN study, respectively. Overall, only a few severe AEs and SAEs occurred in both treatment arms. For severe AEs, SAEs and 'AEs that led to discontinuation of study medication', there was no statistically significant difference between the treatment groups with a high risk of bias. The AEs of special interest 'abnormal injection site reactions' (any type) and 'redness' (both any severity) were reported statistically significantly more frequently in the lonapegsomatropin arm versus the somatropin arm in the heiGHt study, but not in the CT-301-CN study. In summary, regarding morbidity there were favourable results in height (SDS) for lonapegsomatropin versus somatropin in both studies. There was no QoL data and there were mostly no significant differences between the treatment groups in safety outcomes. In absence of blinding, the risk of bias in both studies was high.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/996/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-6495/2024-03-07_AM-RL-XII_Lonapegsomatropin_D-972_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/996/#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/