Emerging treatments
Belumosudil
Belumosudil, a selective oral inhibitor of Rho-associated coiled-coil kinase 2, has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of SSc. A phase 2 double-blind placebo-controlled clinical trial is ongoing in adults with SSc.[68]
Cudetaxestat
Cudetaxestat, an investigational non-competitive, small molecule, reversible inhibitor of autotaxin, has been granted orphan drug designation by the FDA for the treatment of SSc. Recruitment for a phase 1 trial is underway to assess the effect of cudetaxestat on the pharmacokinetics of two approved drugs for idiopathic pulmonary fibrosis (pirfenidone and nintedanib) in healthy volunteers.[69]
Tyrosine kinase inhibitors
Imatinib is a tyrosine kinase inhibitor that is effective for the treatment of chronic myelogenous leukemia and gastrointestinal tumours. Although it has been shown to have anti-fibrotic properties in vitro, preliminary results have been disappointing, due in part to a higher-than-expected incidence of adverse events.[49] Other tyrosine kinase inhibitors are being studied for potential utilisation in SSc.
Pirfenidone
Pirfenidone has been shown to reduce functional decline and disease progression in idiopathic pulmonary fibrosis, but effectiveness in SSc-related interstitial lung disease has not yet been established.[49]
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