Tafamidis (3) – Vyndaqel®
Amyloid Neuropathy
Characteristics
| Start date | 01.12.2020 – Marketing authorisation: 16.11.2011 |
|---|---|
| Resolution | 20.05.2021 |
| INN | Tafamidis |
| Brand name | Vyndaqel® |
| Pharm. company | Pfizer Pharma GmbH |
| G-BA Procedure ID | D-611 |
| ATC code | N07XX08 Other nervous system drugs (N07XX) |
| ICD-10 codes (AIS) | E85.0Non-neuropathic heredofamilial amyloidosis, E85.1Amyloid polyneuropathy (Portuguese), E85.2Heredofamilial amyloidosis, unspecified, E85.4Localized amyloidosis, E85.8Other amyloidosis, E85.9Amyloidosis, unspecified |
| Alpha-ID codes (AIS) | I129348Hereditary transthyretin amyloidosis, I129352Wild-type transthyretin amyloidosis, I24316Amyloidosis, I2490Non-neuropathic heredofamilial amyloidosis, I2491Neuropathic heredofamilial amyloidosis, I66392Localized amyloidosis |
| ORPHAcodes (AIS) | 271861Hereditary transthyretin amyloidosis, 330001Wild-type transthyretin amyloidosis, 69Amyloidosis, |
| DDD | 20 mg O |
| Therapeutic area | Metabolic diseases Amyloidosis Orphan (turnover limit) |
| Reason for procedure |
Reassessment: Orphan turnover exceeded
Original resolution: Tafamidis (1) (07.06.2012) |
| Regulatory status | Exceptional Circumstances |
| Specialty | Bundling |
| Therapeutic indication of the resolution |
|---|
|
Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Adult patients with transthyretin amyloidosis with symptomatic stage 1 polyneuropathy | Patisiran |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (no data submitted) |
|---|---|
|
Study design
(best subpopulation) |
no data submitted |
- Clinical trials
- In its dossier for the assessment of the additional benefit of Tafamidis, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
- Furthermore, no indirect comparisons were presented to address the issues raised in the benefit assessment.
Adult patients with transthyretin amyloidosis and symptomatic stage 1 polyneuropathy
- For adult patients with transthyretin amyloidosis and symptomatic stage 1 polyneuropathy, the additional benefit of Tafamidis compared with the appropriate comparator therapy is not proven.
- In its dossier for the assessment of the additional benefit of tafamidis, the pharmaceutical manufacturer does not present any direct comparative studies against the appropriate comparator therapy.
- Furthermore, no indirect comparisons were submitted to address the issues raised in the benefit assessment.
- Consequently, there are no suitable data available overall for the assessment of the additional benefit of Tafamidis.
- Overall assessment
- On balance, for adult patients with transthyretin amyloidosis and symptomatic stage 1 polyneuropathy, the additional benefit of Tafamidis compared with the appropriate comparator therapy is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
| Tafamidis (4) | Vyndaqel® | Pfizer Pharma GmbH | Amyloid cardiomyopathy | 1,760–1,810 | 100% Indication of considerable additional benefit Orphan (turnover limit) | |
| Tafamidis (3) | Vyndaqel® | Pfizer Pharma GmbH | Amyloid Neuropathy | 230 | 100% additional benefit not proven Orphan (turnover limit) | |
| Tafamidis (2) | Vyndaqel® | Pfizer Pharma GmbH | Amyloid cardiomyopathy |
0
1,630–1,730 |
100% Hint for considerable additional benefit Orphan repealed | |
| Tafamidis (1) | Vyndaqel® | Pfizer Pharma GmbH | Amyloid Neuropathy |
0
40–104 |
100% minor additional benefit Orphan repealed |
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