Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Vosoritid (Achondroplasie, ≥ 2 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: Vosoritide (Voxzogo®) is used for treatment of achondroplasia in patients aged 2 years and older in whom the epiphyses are not yet closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. No information on efficacy and safety in the pivotal population of 2 to 5-year olds could be included in the benefit assessment. The benefit assessment is based on the double-blind, controlled, multicentre, phase 3 study BMN 111-301 in which children and adolescents 5 to 17 years of age with confirmed ACH were randomly assigned in a 1:1 ratio to 15 μg/kg vosoritide and placebo. In the morbidity category, only results for the endpoint "Height" were considered. For this endpoint, there was a statistically significant benefit in favor of vosoritide over placebo with unclear risk of bias. For the study population of 5- to 7-year-olds, no assessments of quality of life were available for the benefit assessment. The results of the PedsQL and the QoLISSY in the age group 8-17 years were only available as descriptive analyses, and therefore group differences could not be assessed. Overall, only minor differences in the occurrence of AEs were observed between the study arms, both numerically in favor and unfavor of vosoritide. Only a few severe AEs and SAEs were observed. The confidence in the results is generally limited due to the small study size. An amendment contains supplemental data on analyses submitted in the course of the hearing procedure, additional analyses regarding three endpoints and results from the uncontrolled extension study to study BMN 111-301.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/745/#english

Year Published: 2022

URL for published report: https://www.g-ba.de/downloads/39-1464-5333/2022-03-18_AM-Rl-XII_Vosoritid_D-737_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/745/#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/