Evinacumab (2) – Evkeeza®
Homozygous familial hypercholesterolemia, ≥ 5 to < 12 years of age
Characteristics
| Start date | 15.01.2024 – Marketing authorisation: 11.12.2023 |
|---|---|
| Resolution | 04.07.2024 |
| INN | Evinacumab |
| Brand name | Evkeeza® |
| Pharm. company | Ultragenyx Germany GmbH |
| G-BA Procedure ID | D-1028 |
| ATC code | C10AX17 Other lipid modifying agents (C10AX) |
| ICD-10 codes (AIS) | E78.0Pure hypercholesterolemia, E78.2Mixed hyperlipidemia, E78.4Other hyperlipidemia, E78.5Hyperlipidemia, unspecified, E78.88, E78.9Disorder of lipoprotein metabolism, unspecified |
| Alpha-ID codes (AIS) | I16606Hyperlipidemia, I16611Hyperlipoproteinuria, I2449Mixed hyperlipidemia, I2459Familial hyperlipidemia, I64600Primary hypercholesterolemia, I94846Dyslipidemia |
| Therapeutic area | Metabolic diseases Hypercholesterolemia |
| Reason for procedure | New therapeutic indication |
| Regulatory status | Exceptional Circumstances |
| Specialty | Bundling Combination therapy |
| Therapeutic indication of the resolution |
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|
Evkeeza is used as an adjuvant therapy to diet and other therapies to lower low density lipoprotein cholesterol (LDL-C) levels for the treatment of adolescents and children aged 5 to < 12 years with homozygous familial hypercholesterolemia (HoFH) |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Children and adolescents aged 5 to < 12 years with homozygous familial hypercholesterolemia in whom dietary and drug options for lipid lowering have been exhausted | - Evolocumab (from 10 years of age) possibly with concomitant lipid-lowering drug therapy, or – LDL apheresis (as an "ultima ratio" for therapy-refractory courses) possibly with concomitant lipid-lowering drug therapy, or – Evolocumab (from 10 years of age) and LDL apheresis (as a last resort for refractory courses), possibly with concomitant lipid-lowering drug therapy |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: Single-arm + no comparison) |
- Clinical trials
- The pharmaceutical manufacturer has submitted the single-arm trial R1500-CL-17100 for the benefit assessment of evinacumab.
Children and adolescents aged 5 to < 12 years with homozygous familial hypercholesterolaemia, in whom dietary and pharmacological options for lowering lipid levels have been exhausted
- The additional benefit is not proven.
- To assess the additional benefit of evinacumab for the treatment of children and adolescents aged 5 to < 12 years with homozygous familial hypercholesterolaemia, in whom dietary and pharmacological options for lowering lipids have been exhausted, the pharmaceutical manufacturer cites the single-arm study R1500-CL-17100.
- Due to the lack of a comparator, the single-arm study R1500-CL-17100 is not suitable for drawing conclusions regarding the additional benefit of evinacumab compared with the appropriate comparator therapy.
Courtesy translation only, please refer to the German original.
Associated procedures
| Evinacumab (1) | Evkeeza® | Ultragenyx Germany GmbH | Homozygous familial hypercholesterolemia, ≥ 12 years of age | 73–80 | 100% additional benefit not proven | |
| Evinacumab (2) | Evkeeza® | Ultragenyx Germany GmbH | Homozygous familial hypercholesterolemia, ≥ 5 to < 12 years of age | 5–6 | 100% additional benefit not proven |
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