Eftrenonacog alfa (2) – Alprolix®
Hemophilia B
Characteristics
| Start date | 01.08.2023 – Marketing authorisation: 12.05.2016 |
|---|---|
| Resolution | 01.02.2024 |
| INN | Eftrenonacog alfa |
| Brand name | Alprolix® |
| Pharm. company | Swedish Orphan Biovitrum GmbH |
| G-BA Procedure ID | D-961 |
| ATC code | B02BD34 Blood coagulation factors (B02BD) |
| ICD-10 codes (AIS) | D67Hereditary factor IX deficiency |
| Alpha-ID codes (AIS) | I27821Hemophilia B |
| ORPHAcodes (AIS) | 98879Hemophilia B |
| Therapeutic area | Hematopoietic diseases Hemophilia (Hemophilia A /Hemophilia B) Orphan (turnover limit) |
| Reason for procedure |
Reassessment: Orphan turnover exceeded
Original resolution: Eftrenonacog alfa (1) (15.12.2016) |
| Therapeutic indication of the resolution |
|---|
|
Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). Alprolix can be used in all age groups. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Patients of all ages with haemophilia B | Recombinant or human plasma-derived coagulation factor IX preparations |
Studies and Results
|
No. of studies
(best subpopulation) |
4 (B-LONG, Kids B-LONG, PUPs B-LONG, B YOND) |
|---|---|
|
Study design
(best subpopulation) |
Single-arm + no comparison |
|
Meta analysis
(best subpopulation) |
no |
- Clinical trials
- In support of this, the pharmaceutical manufacturer cites the four single-arm, non-randomised, uncontrolled clinical studies B-LONG, Kids B-LONG, PUPs B-LONG and B YOND, involving male patients with severe haemophilia B (defined as ≤ 2 IU/dl endogenous factor IX activity).
Patients of all age groups with haemophilia B
- For patients of all age groups with haemophilia B, the additional benefit of eftrenonacog alfa compared with the appropriate comparator therapy is not proven.
- In its dossier for the assessment of the additional benefit of eftrenonacog alfa, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
- The single-arm studies submitted are not suitable for assessing additional benefit due to the lack of comparison with the appropriate comparator therapy.
- Overall, on the basis of the studies submitted, no additional benefit compared with the appropriate comparator therapy can be inferred for patients of all age groups with haemophilia B.
- Overall assessment
- In summary, for patients of all age groups with haemophilia B, the additional benefit of eftrenonacog alfa compared with the appropriate comparator therapy is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
| Eftrenonacog alfa (2) | Alprolix® | Swedish Orphan Biovitrum GmbH | Hemophilia B | 560–720 | 100% additional benefit not proven Orphan (turnover limit) | |
| Eftrenonacog alfa (1) | Alprolix® | Swedish Orphan Biovitrum GmbH | Hemophilia B |
0
580–660 |
100% non-quantifiable additional benefit Orphan repealed |
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