Emicizumab (3) – Hemlibra®
Moderate haemophilia A, without factor VIII inhibitors, with severe bleeding phenotype
Characteristics
| Start date | 01.03.2023 – Marketing authorisation: 23.01.2023 |
|---|---|
| Resolution | 17.08.2023 |
| INN | Emicizumab |
| Brand name | Hemlibra® |
| Pharm. company | Roche Pharma AG |
| G-BA Procedure ID | D-912 |
| ATC code | B02BX06 Other systemic hemostatics (B02BX) |
| ICD-10 codes (AIS) | D66Hereditary factor VIII deficiency |
| Alpha-ID codes (AIS) | I27819Hemophilia A |
| Therapeutic area | Hematopoietic diseases Hemophilia (Hemophilia A /Hemophilia B) |
| Reason for procedure | New therapeutic indication |
| Therapeutic indication of the resolution |
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Emicizumab (Hemlibra) is used as routine prophylaxis of bleeding events in patients with hemophilia A (hereditary factor VIII deficiency) without factor VIII inhibitors with moderate disease (FVIII ≥ 1% and ≤ 5%) with severe bleeding phenotype in all age groups. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Patients with moderate-severe hemophilia A (hereditary factor VIII deficiency, FVIII ≥ 1% and ≤ 5%) and a severe bleeding phenotype without factor VIII inhibitors who are eligible for routine prophylaxis | Plasmatic or recombinant blood coagulation factor VIII preparations, used as routine prophylaxis |
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 HAVEN 6 trial is a single-arm trial that included patients of all age groups with mild (residual Factor VIII activity > 5 % and < 40 %) or moderate (residual Factor VIII activity ≥ 1% and ≤ 5%) hereditary haemophilia A without Factor VIII inhibitors, for whom the investigator deemed prophylaxis to be indicated.
Patients with moderate haemophilia A (hereditary factor VIII deficiency, FVIII ≥ 1% and ≤ 5%) and a severe bleeding phenotype without factor VIII inhibitors, who are eligible for routine prophylaxis
- For patients with moderate haemophilia A (hereditary factor VIII deficiency, FVIII ≥ 1% and ≤ 5%) and a severe bleeding phenotype without factor VIIIinhibitors, who are eligible for routine prophylaxis, the additional benefit of emicizumab as routine prophylaxis compared with the appropriate comparator therapy is not proven.
- Overall, therefore, additional benefit for emicizumab as routine prophylaxis against bleeding episodes in patients with moderate haemophilia A without factor VIII inhibitors and a severe bleeding phenotype is not proven, compared with the appropriate comparator therapy.
- morbidity
- The primary endpoint of the study was treated bleeds, operationalised as the annualised bleeding rate.
- Overall assessment
- In the overall review, additional benefit is not proven for emicizumab in the routine prophylaxis of bleeding episodes in patients with moderate haemophilia A without factor VIII inhibitors and a severe bleeding phenotype, compared with the appropriate comparator therapy.
Courtesy translation only, please refer to the German original.
Associated procedures
| Emicizumab (3) | Hemlibra® | Roche Pharma AG | Moderate haemophilia A, without factor VIII inhibitors, with severe bleeding phenotype | 220–240 | 100% additional benefit not proven | |
| Emicizumab (2) | Hemlibra® | Roche Pharma AG | Hemophilia A, without factor VIII inhibitors | 2,000 | 100% additional benefit not proven | |
| Emicizumab (1) | Hemlibra® | Roche Pharma AG | Hemophilia A | 100 | 50% Hint for non-quantifiable additional benefit |
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