Evinacumab (1) – Evkeeza®
Homozygous familial hypercholesterolemia, ≥ 12 years of age
Characteristics
| Start date | 15.01.2024 – Marketing authorisation: 17.06.2021 |
|---|---|
| Resolution | 04.07.2024 |
| INN | Evinacumab |
| Brand name | Evkeeza® |
| Pharm. company | Ultragenyx Germany GmbH |
| G-BA Procedure ID | D-1027 |
| 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 | Initial assessment |
| Regulatory status | Exceptional Circumstances |
| Specialty | Bundling Combination therapy |
| Therapeutic indication of the resolution |
|---|
|
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 adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH) |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Adolescents aged 12 years and older and adults with homozygous familial hypercholesterolemia for whom dietary and drug options for lipid lowering have been exhausted | - Evolocumab, if necessary with concomitant lipid-lowering drug therapy, or – LDL apheresis (as a last resort for refractory courses), possibly with concomitant lipid-lowering drug therapy, or – Evolocumab 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) |
1 (ELIPSE-HoFH) |
|---|---|
|
Study design
(best subpopulation) |
H2H vs. ACT |
|
Meta analysis
(best subpopulation) |
no |
- Clinical trials
- The randomised, controlled, double-blind ELIPSE-HoFH trial investigated the administration of evinacumab versus placebo, in each case in combination with a low-fat diet and maximally tolerated lipid-lowering therapy, in 65 adults and adolescents aged 12 years and over diagnosed with homozygous familial hypercholesterolaemia (HoFH).
Adolescents aged 12 years and over and adults with homozygous familial hypercholesterolaemia in whom dietary and pharmacological options for lipid-lowering therapy have been exhausted
- The additional benefit is not proven.
- In summary, the additional benefit of evinacumab over the appropriate comparator therapy is not proven in this patient group.
- As a significant proportion of patients in the comparator arm of the presented ELIPSE-HoFH study received neither evolocumab nor LDL apheresis, the appropriate comparator therapy was not adequately implemented in the study, and the study cannot be used to establish additional benefit.
- Furthermore, a longer duration of the ELIPSE-HoFH study would have been advisable for assessing the long-term effects of evinacumab in this indication.
- The single-arm study R1500-CL-1719, presented as supplementary evidence, is not suitable for drawing conclusions regarding the additional benefit of evinacumab compared with the appropriate comparator therapy due to the lack of a comparator.
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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