

Drug discovery
18
drugs
With orphan designations
Overview
Autoimmune Hemolytic Anemia (AIHA) is a rare immune disorder characterized by autoantibody-mediated destruction of red blood cells, leading to anemia. It is classified as warm AIHA (IgG antibodies active at 37°C) or cold AIHA (IgM antibodies reactive at lower temperatures). Symptoms range from fatigue, pallor, and jaundice to severe complications like thrombosis or organ failure. Diagnosis relies on direct antiglobulin (Coombs) testing, with subtyping guiding management [1][6][11].
Burden
One-year mortality: 17.9% (primary AIHA) to 28.4% (secondary AIHA) [2][15].
High healthcare utilization: 48% of severe cases require hospitalization; 55% need transfusions [4][2][15].
Long-term risks: Cardiovascular events, chronic immunosuppression-related infections, and reduced quality of life [2][15][16].
Therapies
First-line: Corticosteroids (prednisone) with response rates of 70–85% [8][12][14].
Refractory/relapsed cases: Rituximab (overall response: 75–90%), splenectomy (long-term remission in ~66%), or immunosuppressants (azathioprine, cyclosporine) [8][12][14].
Cold AIHA: Cold avoidance, rituximab, or complement inhibitors (e.g., sutimlimab under investigation) [6][14][16].
Categories: rare hematological diseases
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
rituximab | antibodies | FDA | 2025-07-18 | — | ODDIFACT SAS |
Bispecific humanized IgG4 BCMA-directed CD3 T cell engager antibody | antibodies | FDA | 2025-06-25 | — | Lakefront Biotherapeutics West LLC |
rilzabrutinib | small molecules | FDA | 2025-02-20 | — | Sanofi US Services Inc. |
Rilzabrutinib | small molecules | EMA | 2025-01-16 | — | Sanofi B.V. |
rituximab | antibodies | FDA | 2023-02-07 | — | Mabion S.A. |
a humanized IgG4 monoclonal antibody, produced in CHO cells, that binds to and inhibits the activated form of the classical complement pathway (CP) specific serine protease, C1s | antibodies | FDA | 2023-01-12 | — | Bioverativ USA Inc |
Parsaclisib | small molecules | EMA | 2022-07-18 | — | Incyte Biosciences Distribution B.V. |
Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s | antibodies | EMA | 2022-07-18 | — | Sanofi B.V. |
rituximab | antibodies | FDA | 2020-11-12 | — | Taxon Therapeutics Ltd. |
parsaclisib | small molecules | FDA | 2020-07-28 | — | Incyte Corporation |
nipocalimab | antibodies | FDA | 2019-12-05 | — | Janssen Research & Development, LLC |
Recombinant complement-specific multimerized human IgG1 Fc | proteins | FDA | 2019-10-21 | — | Gliknik, Inc. |
pegcetacoplan | peptides | FDA | 2019-02-01 | — | Apellis Pharmaceuticals, Inc. |
fostamatinib disodium | small molecules | FDA | 2018-01-31 | — | Rigel Pharmaceuticals, INc. |
sutimlimab-jome [Enjaymo] | antibodies | FDA | 2016-07-27 | 2022-02-04 | Recordati Rare Diseases Inc. |
Humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s [Enjaymo] | antibodies | EMA | 2016-02-17 | 2022-11-23 | Recordati Rare Diseases |
Revimmune | antibodies | FDA | 2011-02-18 | — | Accentia Biopharmaceuticals |
Epoetin alpha | — | FDA | 1989-03-07 | — | R. W. Johnson Pharmaceutical Research Institute |