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)
DDD 20 mg O
Therapeutic area Metabolic diseases Orphan (turnover limit)
Reason for procedure Reassessment: Orphan turnover exceeded
Original resolution: Tafamidis (1) (07.06.2012)
Regulatory status Exceptional Circumstances
Specialty Bundling

Studies and Results

  • 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 Metabolic diseases Amyloid cardiomyopathy 1,760–1,810 100% Indication of considerable additional benefit Orphan (turnover limit)
Tafamidis (3) Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid Neuropathy 230 100% additional benefit not proven Orphan (turnover limit)
Tafamidis (2) Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid cardiomyopathy 0
1,630–1,730
100% Hint for considerable additional benefit Orphan repealed
Tafamidis (1) Vyndaqel® Pfizer Pharma GmbH Metabolic diseases Amyloid Neuropathy 0
40–104
100% minor additional benefit Orphan repealed


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