Drug discovery
18
drugs
With orphan designations
Overview
Limited Systemic Sclerosis (SSc) is a subtype of systemic sclerosis characterized by skin fibrosis restricted to distal extremities and face, often presenting as CREST syndrome (calcinosis, Raynaud’s phenomenon, esophageal dysmotility, sclerodactyly, telangiectasia). It features slow progression but is frequently complicated by pulmonary hypertension, with preserved survival compared to diffuse SSc [1][6][16][19].
Burden
Economic: Annual healthcare costs exceed controls by ~$12,820, driven by frequent hospitalizations and specialty care [4][9].
Morbidity: Skin fibrosis, digital ulcers, and pulmonary hypertension impair quality of life; 30% develop interstitial lung disease [5][14][16].
Survival: 10-year survival is 84–92%, but pulmonary hypertension reduces 3-year survival to 50% [1][6][19].
Therapies
Vasodilation: Calcium channel blockers (e.g., nifedipine) for Raynaud’s; endothelin receptor antagonists (e.g., bosentan) or PDE5 inhibitors (e.g., sildenafil) for pulmonary hypertension [1][3][18].
Symptom management: PPIs for gastroesophageal reflux, immunosuppressants (e.g., methotrexate) for early skin involvement, and annual echocardiography/pulmonary function tests for complication monitoring [1][6][16].
Emerging therapies: Rituximab (anti-CD20) and tocilizumab (anti-IL6) show promise in early trials for fibrosis and lung dysfunction [3][13].
Categories: rare cardiac diseases, rare renal diseases, rare respiratory diseases, rare skin diseases, rare systemic and rheumatological diseases, rare systemic or rheumatologic diseases of childhood, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
2-[1-(3-{6-[(1E)-(hydroxyimino)methyl]-5-methyl-4-oxo-7-propyl-3H,4H-pyrrolo[2,1-f][1,2,4]triazin-2-yl}-4-propoxybenzenesulfonyl)piperidin-4-yl]ethyl nitrate | small molecules | FDA | 2025-08-11 | — | Topadur Pharma AG |
Iloprost | small molecules | EMA | 2025-06-20 | — | MWB Consulting |
artesunate | small molecules | FDA | 2025-01-31 | — | Artasome Therapeutics, LLC |
Avenciguat | small molecules | EMA | 2024-11-11 | — | Boehringer Ingelheim International GmbH |
anti-ephrinB2 humanized IgG1 monoclonal antibody | antibodies | FDA | 2024-10-18 | — | Mediar Therapeutics, Inc. |
autologous anti-CD19 CAR T cell immunotherapy | cell therapies | FDA | 2024-09-20 | — | Kyverna Therapeutics, Inc. |
Ziritaxestat | small molecules | EMA | 2020-01-09 | — | Lakefront Biotherapeutics |
Romilkimab | antibodies | EMA | 2020-01-09 | — | Sanofi-Aventis Groupe |
Lenabasum | small molecules | EMA | 2017-01-12 | — | Pharma Gateway AB |
Nintedanib esylate [Ofev] | small molecules | EMA | 2016-08-29 | — | Boehringer Ingelheim International GmbH |
Autologous adipose tissue-derived stromal vascular fraction | cell therapies | EMA | 2016-04-28 | — | Scendea (NL) B.V. |
Lanifibranor [IVA 337] | small molecules | EMA | 2014-11-19 | — | Inventiva S.A.S. |
Riociguat [Adempas] | small molecules | EMA | 2014-07-29 | — | Bayer AG |
Terguride | small molecules | EMA | 2013-02-08 | — | [INACTIVE] High Tech Participations GmbH |
TERGURIDE HYDROGENMALEATE | small molecules | EMA | 2013-01-24 | — | Medac Gesellschaft für klinische Spezialpräparate mbH |
Pomalidomide [Imnovid] | small molecules | EMA | 2012-04-26 | — | Celgene Europe Limited |
Metelimumab | antibodies | EMA | 2002-02-04 | — | [INACTIVE] Sanofi B.V. |
Halofuginone hydrobromide | small molecules | EMA | 2001-12-11 | — | PPD Global Limited |