Nonacog beta pegol (2) – Refixia®
Hemophilia B, < 12 years
Characteristics
| Start date | 01.09.2023 – Marketing authorisation: 04.08.2023 |
|---|---|
| Resolution | 15.02.2024 |
| INN | Nonacog beta pegol |
| Brand name | Refixia® |
| Pharm. company | Novo Nordisk Pharma GmbH |
| G-BA Procedure ID | D-969 |
| ATC code | B02BD36 Blood coagulation factors (B02BD) |
| ICD-10 codes (AIS) | D67Hereditary factor IX deficiency |
| Alpha-ID codes (AIS) | I27821Hemophilia B |
| Therapeutic area | Hematopoietic diseases Hemophilia (Hemophilia A /Hemophilia B) |
| Reason for procedure | New therapeutic indication |
| Therapeutic indication of the resolution |
|---|
|
Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency) |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Patients aged < 12 years with haemophilia B | Recombinant or human plasma-derived coagulation factor IX preparations |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (no data submitted) |
|---|---|
|
Study design
(best subpopulation) |
no data submitted |
- Clinical trials
- In addition, the pharmaceutical manufacturer has submitted the single-arm studies forming the basis for marketing authorisation: Paradigm 5 (previously treated male patients aged ≤ 12 years) and Paradigm 6 (previously untreated male patients aged < 6 years) involving patients with severe haemophilia B (≤ 2 % Factor IX activity).
Patients aged < 12 years with haemophilia B
- For patients aged < 12 years with haemophilia B, the additional benefit of nonacog beta pegol compared with the appropriate comparator therapy is not proven.
- In its dossier for the assessment of the additional benefit of Nonacog beta pegol, 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 can be inferred for patients aged < 12 years with haemophilia B compared with the appropriate comparator therapy.
- Overall assessment
- In the overall review, the additional benefit of Nonacog beta pegol compared with the appropriate comparator therapy is not proven for patients aged < 12 years with haemophilia B.
Courtesy translation only, please refer to the German original.
Associated procedures
| Nonacog beta pegol (2) | Refixia® | Novo Nordisk Pharma GmbH | Hemophilia B, < 12 years | 100–110 | 100% additional benefit not proven | |
| Nonacog beta pegol (1) | Refixia® | Novo Nordisk Pharma GmbH | Hemophilia B, ≥ 12 years | 500–570 | 100% additional benefit not proven |
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