Table 3 |.
Selected treatments approved or in development for fibrosis affecting various organs
| Treatment type | Drug or procedure | Ref. or trial identifier |
|---|---|---|
| Lung | ||
| Immunosuppression | Approved: oral mycophenoLate mofetiL, oral or intravenous cyclophosphamide, rituximab (CD20 inhibitor), tociiizumab (IL-6 inhibitor) |
301–303,349 NCT01933334, NCT03068234, NCT03856853, NCT03221257 |
| Antifibrotic therapy | Approved: nintedanib, pirfenidone In development: nerandomiLast (PDE4B inhibitor), bexotegrast (avβ6 and avβ1 integrin inhibitor), admiLparant (LPA1 receptor inhibitor), treprostiniL (prostacyclin analogue) |
139,140,142 NCT06806592, NCT04396756136, NCT06003426, NCT04905693 |
| Other interventions | Lung transplantation, oxygen, inhaled treprostiniL (as treatment for pulmonary hypertension related to fibrotic interstitial Lung disease) | 205,209,350 |
| Gut | ||
| Immunosuppression | None | NA |
| Antifibrotic therapy | In development: AGMB-129 (topical ALK5 inhibitor) | NCT05843578 |
| Other interventions | Approved: surgical resection, surgical strictureplasty, endoscopic baLLoon dilation | 351,352 |
| Kidney | ||
| Immunosuppression | None | NA |
| Antifibrotic therapy | In development: pirfenidone, nintedanib, runcaciguat (sGC activator) | 280,281 |
| Other interventions | Approved: dapagLifLozin, empagLifLozin (both SGLT2 inhibitors), finerenone (mineraLocorticoid receptor antagonist); muLtipLe RAAS inhibitors (angiotensin II receptor bLockers or angiotensin-converting enzyme inhibitors) have shown positive effects on end points of kidney function but Lack direct antifibrotic effects In deveLopment: zibotentan, atrasentan (both endotheLin receptor antagonists), pentoxyfiLLine, diaLysis and kidney transpLantation |
353–358 |
| Skin (SSc) | ||
| Immunosuppression | Approved: oraL mycophenoLate mofetiL; intravenous or oraL cycLophosphamide, rituximab (CD20 inhibitor); high-dose chemotherapy with haematopoietic stem cell transpLantation In deveLopment: tociLizumab (IL-6 inhibitor), Lenabasum (CB2 receptor agonist), CAR-T ceLL therapy |
301–304,359–362 151,363–368 |
| Antifibrotic therapy | In deveLopment: fasudiL, reLaxin, pravastatin (aLL three targeting Rho–ROCK fibrobLast mechanosignaLLing); QAX576 (IL-13 inhibitor), SAR156597 (IL-4/IL-13 inhibitor) |
110
NCT00704665 NCT01268202 NCT00581997 NCT02921971 |
| Other interventions | None | NA |
| Liver | ||
| Immunosuppression | None | NA |
| Antifibrotic therapy | In deveLopment: simtuzumab (LOXL2 inhibitor), QAX576 (IL-4/IL-13 inhibitor) |
369
NCT01266135 |
| Other interventionsa | Approved: resmetirom (thyroid hormone receptor β agonist) In deveLopment: GSK-233072 (ASBT inhibitor); cenicriviroc (CCR2/5 receptor antagonist); semagLutide (GLP1 receptor agonist), tirzepatide (GLP1/GIP receptor agonist); obetichoLic acid (FXR agonist); Lanifibranor (pan-PPAR agonist); efruxifermin (Long-acting FGF21 anaLogue) |
333,369,331,332 NCT03900429 |
ALK5, activin receptor-like kinase 5; ASBT, apical sodium-bile acid transporter; CAR, chimeric antigen receptor; CB2 receptor, cannabinoid receptor type 2; CCR2; CC chemokine receptor 2; FGF21; fibroblast growth factor 21; FXR, farnesoid X receptor; GLP1, glucagon-like peptide 1; GIP, gastric inhibitory polypeptide; LPA, lysophosphatidic acid; LOXL2, lysyl oxidase-like 2; NA, not applicable; PDE4B, phosphodiesterase 4B; PPAR, peroxisome proliferator-activated receptor; RAAS, renin-angiotensin-aldosterone system; ROCK, Rho-associated protein kinase; sGC, soluble guanylate cyclase; SGLT2, sodium-glucose transporter 2; SSc, systemic sclerosis.
Most drugs in clinical trials for fibrotic liver diseases (in particular, metabolic dysfunction-associated steatohepatitis (MASH)) aim to reduce the metabolic and/or inflammatory impact of the disease and are not direct antifibrotics.