Patient group
Erwachsene mit Morbus Pompe (Mangel an saurer α-Glucosidase [GAA]) der späten Verlaufsform
completed2023-08-01D series
Cipaglucosidase alfa
Morbus Pompe, Kombination mit Miglustat
Open official case page ↗
- Brand
- Pombiliti
- Manufacturer
- Amicus Therapeutics GmbH
G-BA action
final resolution
2024-02-01
Arzneimittel-Richtlinie/Anlage XII: Cipaglucosidase alfa (Morbus Pompe, Kombination mit Miglustat)
Evidence-checked summary
The G-BA found a hint of minor added benefit for Erwachsene mit Morbus Pompe (Mangel an saurer α-Glucosidase [GAA]) der späten Verlaufsform. The appropriate comparator was Alglucosidase alfa. The G-BA issued a final resolution on 2024-02-01. The official page states that it took effect as follows: 01.02.2024. The page lists Arzneimittel-Richtlinie, Anlage XII: Nutzenbewertung nach § 35a SGB V as the normative target.
This is the latest validated public action attached to this case. It is separate from the IQWiG assessment result.
Final benefit result
G-BA result by patient group
Official case record
What happened, in source order
No inferred stages
Consultation record
Oral hearing and Wortprotokoll
2023-12-11
50 parsed turns
Oral hearing: Cipaglucosidase alfa (Morbus Pompe, Kombination mit Miglustat)
Official Wortprotokoll · 10 pages
IQWiG comparison
Assessment record versus G-BA action
IQWiG assessment findings and the later G-BA action are separate records. Open both to compare them.
A23-79
matched
Cipaglucosidase alfa (Pompe disease) – Benefit assessment according to § 35a Social Code Book (SGB) V
Official assessment PDF · 90 pages
G-BA decisions
Distinct actions and documents
2024-02-01
final resolution
Arzneimittel-Richtlinie/Anlage XII: Cipaglucosidase alfa (Morbus Pompe, Kombination mit Miglustat)
The G-BA found a hint of minor added benefit for Erwachsene mit Morbus Pompe (Mangel an saurer α-Glucosidase [GAA]) der späten Verlaufsform. The appropriate comparator was Alglucosidase alfa. The G-BA issued a final resolution on 2024-02-01. The official page states that it took effect as follows: 01.02.2024. The page lists Arzneimittel-Richtlinie, Anlage XII: Nutzenbewertung nach § 35a SGB V as the normative target.
3 official documents · 4 cited claims
Official sources
Case document package
official case pageOfficial G-BA case pageOpen ↗iqwig addendumAddendum zur Nutzenbewertung des IQWiGOpen ↗annex xii current versionCipaglucosidase alfaOpen ↗comparator informationInformationen zur zweckmäßigen VergleichstherapieOpen ↗hearing transcriptWortprotokollOpen ↗decision textBeschlusstextOpen ↗decision rationaleTragende Gründe zum BeschlussOpen ↗summary documentationZusammenfassende DokumentationOpen ↗iqwig assessmentNutzenbewertung IQWiGOpen ↗
Generic graph relations · 6
HAS ASSESSMENTCipaglucosidase alfaHAS ASSOCIATED DECISIONArzneimittel-Richtlinie/Anlage XII: Cipaglucosidase alfa (Morbus Pompe, Kombination mit Miglustat)HAS HEARINGOral hearing: Cipaglucosidase alfa (Morbus Pompe, Kombination mit Miglustat)OWNS DOCUMENTAddendum zur Nutzenbewertung des IQWiGOWNS DOCUMENTCipaglucosidase alfaOWNS DOCUMENTInformationen zur zweckmäßigen Vergleichstherapie