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New HTA Decisions in Germany
December 2020
Drug name
KALYDECO® (ivacaftor)
Company
Vertex Pharmaceuticals
Decision date
01/10/2020
Therapeutic area
Respiratory conditions
Therapeutic sub area
Cystic fibrosis
G-BA decision date
15/12/2020
Orphan Drug?
Yes
Decision
Additional benefit not quantifiable
Indication
Patients aged 6 months to <18 years old with cystic fibrosis who have a R117H mutation of the CFTR gene. Main comparator: BCS
Decision Detail
Main study: VX11-770-110 Main driver of decision: No appropriate data were presented to inform a benefit decision concerning patients aged 6 months to <6 years. For patients aged 6 years to <18 years, data did not show significant positive or negative effects of ivacaftor compared to the comparator.
Summary
IQWiG concluded that additional benefit was not proven in the use of ivacaftor for treatment of cystic fibrosis in patients aged 6 months to <18 years with a R117H mutation of the CFTR gene. As this is an orphan drug, the G-BA will make the final benefit assessment.