Avatrombopag (2) – Doptelet®

Immune thrombocytopenia (ITP)

Characteristics

Start date 01.04.2021 – Marketing authorisation: 20.06.2019
Resolution 16.09.2021
INN Avatrombopag
Brand name Doptelet®
Pharm. company Swedish Orphan Biovitrum GmbH
G-BA Procedure ID D-649
ATC code B02BX08 Other systemic hemostatics (B02BX)
ICD-10 codes (AIS) D69.3Hemorrhagic (thrombocytopenic) purpura, D69.40, D69.41Evans syndrome
Alpha-ID codes (AIS) I1922Chronic idiopathic thrombocytopenia, I80017Primary thrombocytopenia, I86898Transfusion-refractory congenital thrombocytopenia
DDD 20 mg O
Therapeutic area Hematopoietic diseases Immune thrombocytopenia (ITP), Thrombocytopenia
Reason for procedure Initial assessment
Specialty Bundling

Therapeutic indication of the resolution

Doptelet is used for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who do not respond to other therapies (e.g., corticosteroids, immunoglobulins).

Subpopulation Indication Comparator
Adults with primary chronic immune thrombocytopenia (ITP) who do not respond to other therapies (e.g. corticosteroids, immunoglobulins). Eltrombopag or Romiplostim

Studies and Results

No. of studies
(best subpopulation)
0 (Data not accepted)
Study design
(best subpopulation)
Data not accepted (Dossier: H2H vs. ACT)

  • Clinical trials
    • Study 302 is a randomised, double-blind Phase III trial comparing avatrombopag with placebo in patients with ITP who have previously received at least one course of treatment.
    • The supplementary Study 305 is a double-blind, randomised Phase III trial comparing avatrombopag with eltrombopag in adult patients with pre-treated primary chronic ITP.

Adults with primary chronic immune thrombocytopenia (ITP) who do not respond to other treatments (e.g. corticosteroids, immunoglobulins)

  • An additional benefit is not proven.
  • In summary, based on the data provided, no conclusions can be drawn regarding the additional benefit of avatrombopag. The additional benefit of avatrombopag compared with the appropriate comparator therapy is not proven.
  • Overall assessment
    • Study 302 compares avatrombopag with placebo. As Study 302 did not compare avatrombopag with the specified appropriate comparator therapy, the study is not suitable for assessing additional benefit.
    • Study 305 compares avatrombopag with eltrombopag. The study had to be terminated prematurely due to recruitment problems and does not meet the minimum study duration of 24 weeks considered necessary for chronic conditions. The data from the study are not suitable for assessing additional benefit.
    • The further analyses (network meta-analysis, comparison of data from individual studies) are not suitable for assessing additional benefit due to significant methodological shortcomings and selective presentation of endpoints.

Courtesy translation only, please refer to the German original.

Associated procedures

Avatrombopag (1) Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Thrombocytopenia in chronic liver disease 1,790–24,130 100% additional benefit not proven
Avatrombopag (2) Doptelet® Swedish Orphan Biovitrum GmbH Hematopoietic diseases Immune thrombocytopenia (ITP) 4,260–10,830 100% additional benefit not proven


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