Original Title: Arzneimittel-Richtlinie/Anlage XII: Eladocagene Exuparvovec (Aromatische-L-Aminosäure-Decarboxylase-(AADC)-Mangel, ≥ 18 Monate)

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: Eladocagene exuparvovec is approved for the treatment of patients aged 18 months and older with a clinically, molecularly and genetically confirmed diagnosis of AADC deficiency with a severe phenotype. The benefit assessment of eladocagene exuparvovec is based on the pivotal studies AADC-010, AADC-011 and AADC-CU/1601, which are monocentric, single-arm phase 1/2 studies investigating the safety and efficacy of eladocagene exuparvovec in children with AADC deficiency. Due to the small sample size and the lack of a valid comparison, a conclusive assessment of effects on mortality is not possible. There are positive changes in the total score of the PDMS-2 and AIMS as well as the cognitive scale and the language scales of the BSID-III. Due to the lack of valid comparison, the small sample size, the analysis as absolute values in the context of possible age effects and the partly unknown scale ranges, a conclusive interpretation of the observed effects is not possible. No results on quality of life were presented. Numerous adverse events and serious adverse events were observed, which could also include events from the morbidity category. A conclusive assessment on safety is not possible due to the lack of a valid comparison and the small sample size.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/868/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-5864/2023-02-02_AM-RL-XII_Eladocagene-Exuparvovec_D-856_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/868/#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/