Drug discovery
122
drugs
With orphan designations
Overview
Soft tissue sarcoma (STS) comprises over 50 mesenchymal malignancies, typically arising in extremities, trunk, or retroperitoneum. Diagnosis requires biopsy and advanced imaging (MRI/CT) [1][6]. Treatment involves multidisciplinary care, with limb-sparing surgery combined with radiation therapy as standard for localized disease [3][8]. Chemotherapy and targeted therapies (e.g., imatinib for GISTs) are reserved for advanced/metastatic cases [5][18]. Prognosis depends on histology, grade, and stage.
Population
Annual incidence: ~3.4–4.5/100,000, rising sharply after age 50; median age at diagnosis 62 [2][10]
Affects males more frequently (male:female ratio ~1.3:1), with higher rates in non-Hispanic whites [2][7][10]
Risk factors: Radiation exposure, genetic syndromes (NF1, Li-Fraumeni), and chemical carcinogens [6][12]
Burden
Represents 1% of adult cancers but 15% of pediatric malignancies [19]; ~13,520 new U.S. cases projected for 2025 [10]
5-year survival: 66% for localized STS vs. 16% for metastatic disease [10]; mortality rates declining (EAPC -0.81% globally) [4]
Healthcare disparities: Non-referral to sarcoma centers doubles local recurrence risk (39% vs. 19%) [3][17]
Therapies
Localized disease: Wide surgical resection ± neoadjuvant/adjuvant radiation (5-year local control >85%) [3][13]
Advanced disease: Doxorubicin-based chemotherapy (response rate 15–25%); histology-specific agents (e.g., trabectedin, pazopanib) [5][18]
Emerging approaches: Immunotherapy (checkpoint inhibitors in clinical trials) and precision radiotherapy (SBRT) [5][8]
Categories: rare neoplastic diseases
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
alpha-emitting radiolabeled small molecule that selectively binds to fibrobast activation protein-alpha (FAP) ([Ac-225]RTX-2358) | small molecules | FDA | 2026-06-09 | — | Ratio Therapeutics, Inc. |
recombinant, single-chain, human Interleukin-12 linked to a proprietary fully-human A10m3-albumin binding domain | proteins | FDA | 2026-05-28 | — | Guidant BioTherapeutics Inc. |
legubicin | small molecules | FDA | 2026-05-18 | — | Catalysis Therapeutics Inc. |
eftilagimod alfa | proteins | FDA | 2026-04-12 | — | Immutep S.A.S |
Zika virus strain ZIKV-Nicaragua/2016 | other | FDA | 2026-03-17 | — | The Nemours Foundation |
an antibody drug conjugate (ADC) composed of an anti-uPARAP humanized antibody, conjugated to a topoisomerase I inhibitor | antibodies | FDA | 2026-03-16 | — | Adcendo ApS |
a humanized MUC18-targeting IgG1 antibody conjugated with exatecan, a topoisomerase I inhibitor | antibodies | FDA | 2026-03-05 | — | Multitude Therapeutics Inc. |
3-((4-((4-amino-2-butyl-1H-imidazo[4,5-c]quinolin-1-yl)oxy)butyl)(3- (dimethylamino)propyl)amino)-4-(heptadecylamino)cyclobut-3-ene-1,2-dione | small molecules | FDA | 2026-02-27 | — | Canwell Pharma, Inc. |
(+)N-hydroxy-N(methylacylfulvene)urea | other | FDA | 2026-01-15 | — | Lantern Pharma Inc. |
a synthetic drug conjugate comprised of a ligand targeting CD206, a polysaccharide backbone, a valine citrulline linker, and a p-aminobenzyl carbamate self-immolative spacer with the toxin monomethyl auristatin E | other | FDA | 2025-10-16 | — | Resolute Science, Inc. |
16 base single stranded peptide nucleic acid oligonucleotide - 7 amino acids peptide | oligonucleotides | FDA | 2025-10-16 | — | Biogenera SpA |
Varegacestat | small molecules | EMA | 2025-08-22 | — | Somerville Development Partners B.V. |
anagrelide | small molecules | FDA | 2025-07-22 | — | Sartar Therapeutics Oy |
Doxorubicin, liposomal, pegylated | small molecules | EMA | 2025-05-22 | — | InnoMedica Deutschland GmbH |
doxorubicin hydrochloride encapsulated in liposomes | small molecules | FDA | 2025-04-15 | — | InnoMedica Schweiz AG |
recombinant vesicular stomatitis virus with a target gene of NY-ESO-1 | other | FDA | 2025-03-27 | — | Joint Biosciences (SH) Ltd. |
encapsulated doxorubicin in a thermosensitive liposome | small molecules | FDA | 2025-03-21 | — | Thermosome GmbH |
small-molecule inhibitor specifically targeting Galectin-1 | small molecules | FDA | 2025-03-10 | — | Kibio Inc |
oncolytic swine glyco-humanized polyclonal antibody | antibodies | FDA | 2025-03-05 | — | Xenothera |
N-hydroxy-N-(methylacylfulvene)urea | small molecules | FDA | 2024-10-28 | — | Lantern Pharma Inc. |
padnarsertib | small molecules | FDA | 2024-07-08 | — | Karyopharm Therapeutics Inc. |
elraglusib | small molecules | FDA | 2024-07-03 | — | Actuate Therapeutics, Inc. |
N-(5-cyano-4-((2-methoxyethyl)amino)pyridin-2-yl)-7-formyl-6-((4-methyl-2- oxopiperazin-1-yl)methyl)-3,4-dihydro-2,4-methano-1,8-naphthyridine-1(2H)- carboxamide 2-hydroxypropane-1,2,3-tricarboxylate | small molecules | FDA | 2024-07-01 | — | BroadenBio Co., Ltd. |
(R)-3-(((6-(methyl(phenyl)amino)-1,2,3,4-tetrahydronaphthalen-1-yl)methyl)amino)isonicotinic acid | small molecules | FDA | 2024-06-11 | — | Tachyon Therapeutics, Inc. |
bispecific Fc fusion protein containing a CD80 ectodomain and an IL-2v3 moiety | antibodies | FDA | 2024-06-10 | — | GI Innovation, Inc. |
(S)-N-(1-(3-fluoro-2'-methoxy-[1,1'-biphenyl]-4-yl)-2-oxopiperidin-3-yl)-5-(pyridin-2-yl) thiophene-2-sulfonamide | small molecules | FDA | 2024-04-08 | — | Amira Therapeutics, S.L. |
Tigilanol Tiglate | small molecules | FDA | 2024-02-08 | — | QBiotics Group Limited |
Small molecule inhibitor of PRMT5 | small molecules | FDA | 2023-12-21 | — | Tango Therapeutics, Inc. |
(S)-N-(1-(3-Fluoro-2'-methoxy-[1,1'-biphenyl]-4-yl)-2-oxopiperidin-3-yl)-5-(pyridin-2-yl)thiophene-2-sulfonamide | small molecules | EMA | 2023-12-13 | — | Amira Therapeutics S.L. |
Brigimadlin | small molecules | EMA | 2023-06-20 | — | Boehringer Ingelheim International GmbH |
Idronoxil | small molecules | EMA | 2023-05-22 | — | CATS Consultants GmbH |
Lurbinectedin | small molecules | EMA | 2023-04-21 | — | Pharma Mar S.A. |
3-(5-cyano-4-(cyclopropylamino)pyridin-2-yl)-1-(6-formyl-5-((4-methyl-2-oxopiperazin-1-yl)methyl)pyridin-2-yl)-1-methylurea | small molecules | FDA | 2023-03-27 | — | Wuxi Abbisko Biomedical Technology Co., Ltd. |
Single Protein Encapsulated Doxorubicin | proteins | FDA | 2022-11-15 | — | Sunstate Biosciences LLC |
N-[(2R)-1-[(2S)-2-[(3-hydroxy-2-methyl-6-{[(3S)-3,5,12-trihydroxy-3-(2-hydroxyacetyl)-10-methoxy-6,11-dioxo-1,2,3,4,6,11-hexahydrotetracen-1-yl]oxy}oxan-4-yl)carbamoyl]pyrrolidin-1-yl]-1-oxopropan-2-yl]pyridine-4-carboxamide | small molecules | FDA | 2022-09-01 | — | Avacta Life Sciences Ltd. |
Doxorubicin | small molecules | EMA | 2022-06-21 | — | Thermosome GmbH |
cis-diamminedichloroplatinum(II) (CDDP), vinblastine sulfate, 8-((2-hydroxybenzoyl)amino)octanoate (SHAO-FA) | small molecules | FDA | 2022-06-13 | — | Intensity Therapeutics, Inc. |
Idronoxil | small molecules | FDA | 2022-03-17 | — | Noxopharm Limited |
small molecule composed of a ligand binding moiety to BRD9, a chemical linker, and a ligand binding moiety to the cereblon (CRBN) E3 ligase | small molecules | FDA | 2022-03-08 | — | C4 Therapeutics, Inc. |
Unesbulin | small molecules | EMA | 2021-12-10 | — | PTC Therapeutics International Limited |
Pegylated Liposomal Alendronate with Doxorubicin | small molecules | FDA | 2021-11-08 | — | InnoMedica Schweiz AG |
Biocompatible polymeric PLGA nanofiber membrane containing the active substance 7-ethyl-10-hydroxycamptothecin | small molecules | FDA | 2021-10-13 | — | Cebiotex S.L. |
Polymeric polyethylene glycol-phosphatidyl ethanolamine (PEG-PE) micelles containing Curcumin C3 complex (CUR) and doxorubicin hydrochloride (DOX) | small molecules | FDA | 2021-09-27 | — | Immix Biopharma, Inc |
IL13 E13K mutation linked to portion of mutated pseudomonas exotoxin | proteins | FDA | 2021-08-03 | — | Targepeutics |
sotigalimab | antibodies | FDA | 2021-08-03 | — | Pyxis Oncology, Inc. |
envafolimab | antibodies | FDA | 2021-06-28 | — | Tracon Pharmaceuticals, Inc. |
humanized IgG1anti-AXL-antibody conjugated to monomethyl auristatin E | antibodies | FDA | 2021-03-01 | — | BioAtla, Inc. |
Talabostat | small molecules | FDA | 2021-01-26 | — | BioXcel Therapeutics |
ilixadencel | cell therapies | FDA | 2021-01-25 | — | Mendus AB |
liposomal annamycin | small molecules | FDA | 2020-12-28 | — | Moleculin Biotech, Inc. |
4-((3'R,4'S,5'R)-6''-chloro-4'-(3-chloro-2-fluorophenyl)-1'-ethyl-2''-oxodispiro[cyclohexane-1,2'-pyrrolidine-3',3''-indoline]-5'-carboxamido)bicyclo [2.2.2]octane-1-carboxylic acid | small molecules | FDA | 2020-10-28 | — | Ascentage Pharma Group Inc. |
atezolizumab [Tecentriq] | antibodies | FDA | 2020-10-08 | 2022-12-09 | Genentech, Inc. |
Volasertib | small molecules | FDA | 2020-10-08 | — | Oncoheroes Biosciences Inc. |
Toripalimab | antibodies | FDA | 2020-09-15 | — | TopAlliance Biosciences, Inc. |
Autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4 | cell therapies | EMA | 2020-06-04 | — | Brancaster Pharma Ireland Limited |
5-fluoro-2-(6-fluoro-2-methyl-1H-benzo[d]imidazole-1-yl)-N4-(4-(trifluoromethyl)phenyl)pyrimidine-4,6-diamine | small molecules | FDA | 2020-02-13 | — | PTC Therapeutics, Inc. |
Camsirubicin | small molecules | EMA | 2019-11-13 | — | Monopar Therapeutics SARL |
4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide | small molecules | EMA | 2019-10-17 | — | Boyd Consultants Limited |
Nirogacestat [Ogsiveo] | small molecules | EMA | 2019-10-17 | 2025-08-18 | Merck Europe B.V. |
Paclitaxel | small molecules | EMA | 2019-10-17 | — | Boyd Consultants Limited |
afamitresgene autoleucel [Tecelra] | cell therapies | FDA | 2019-08-26 | 2024-08-01 | USWM CT, LLC |
7-ethyl-10-hydroxycamptothecin | small molecules | EMA | 2019-07-25 | — | Cebiotex S.L. |
Vinorelbine tartrate | small molecules | EMA | 2019-01-11 | — | TLC Biopharmaceuticals B.V. |
Fibromun | proteins | FDA | 2018-12-18 | — | Philogen S.p.A. |
conditionally active biologics-anti-ROR2-antibody drug conjugate consisting of a humanized IgG1 antibody specific for ROR2 tyrosine kinase conjugated to a peptide linker coupled with monomethyl auristatin E | antibodies | FDA | 2018-12-18 | — | BioAtla LLC |
1-[[[4-(4-fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride | small molecules | EMA | 2018-02-22 | — | CATS Consultants GmbH |
epirubicin-conjugated polymer micelles | small molecules | FDA | 2017-07-13 | — | NanoCarrier Co., Ltd |
liposomal vinorelbine | small molecules | FDA | 2017-07-13 | — | Taiwan Liposome Company, Ltd. |
Anlotinib | small molecules | FDA | 2017-06-19 | — | Advenchen Laboratories, LLC |
tazemetostat | small molecules | FDA | 2017-06-15 | 2020-01-23 | Epizyme, Inc. |
Milademetan tosilate monohydrate [DS-3032b] | small molecules | EMA | 2017-03-20 | — | S-cubed Pharmaceutical Services ApS |
Propranolol | small molecules | EMA | 2016-12-12 | — | The Anticancer Fund |
Crenolanib besylate | small molecules | EMA | 2016-10-14 | — | Arog Pharmaceuticals Europe Ltd |
A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format | antibodies | EMA | 2016-10-14 | — | Philogen S.p.A. |
Autologous CD4+ and CD8+ T-cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1 | cell therapies | EMA | 2016-07-14 | — | Brancaster Pharma Ireland Limited |
16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7 aminoacid peptide | oligonucleotides | EMA | 2016-07-14 | — | Biogenera SpA |
selinexor | small molecules | FDA | 2016-07-07 | — | Karyopharm Therapeutics, Inc. |
combinatorial regimen of LV305 (lentiviral vector encoding NY-ESO-1 gene) and G305 (NY-ESO-1 recombinant protein plus GLA-SE) | gene therapies | FDA | 2016-05-02 | — | Immune Design Corp. |
Carotuximab | antibodies | EMA | 2016-04-28 | — | Tracon Pharma International Limited |
letetresgene autoleucel | cell therapies | FDA | 2016-03-28 | — | USWM CT, LLC |
Sindbis virus envelope pseudotyped lentiviral vector encoding New York esophageal squamous cell carcinoma-1 | gene therapies | EMA | 2016-03-21 | — | [INACTIVE] Immune Design Limited |
New York esophageal squamous cell carcinoma 1 | cell therapies | EMA | 2016-03-21 | — | [INACTIVE] Immune Design Limited |
Human/Murine Chimeric Monoclonal antibody to Endoglin | antibodies | FDA | 2016-01-20 | — | Tracon Pharmaceuticals, Inc. |
Larotrectinib sulfate [Vitrakvi] | small molecules | EMA | 2016-01-11 | — | Bayer AG |
dendritic-cell targeting, lentiviral vector encoding the NY-ESO-1 gene | gene therapies | FDA | 2016-01-06 | — | Immune Design Corp. |
recombinant NY-ESO-1 protein mixed with glucopyranosyl lipid A | combination | FDA | 2016-01-06 | — | Immune Design Corp. |
5-imino-13-deoxydoxorubicin HCl | small molecules | FDA | 2015-12-31 | — | Monopar Therapeutics, Inc. |
Larotrectinib | small molecules | FDA | 2015-08-31 | — | Bayer HealthCare Pharmaceuticals Inc. |
Olaratumab [Lartruvo] | antibodies | EMA | 2015-02-12 | — | Eli Lilly Nederland B.V. |
olaratumab [Lartruvo] | antibodies | FDA | 2014-10-09 | 2016-10-19 | Eli Lilly and Company |
Aldoxorubicin | small molecules | EMA | 2014-03-26 | — | Pharma Gateway AB |
granulocyte-macrophage colony stimulating factor-coding oncolytic adenovirus, Ad5/3-D24-GMCSF | gene therapies | FDA | 2013-07-24 | — | Targovax Solutions AS, a subsidiary of Targovax ASA |
Adenovirus serotype 5/3 coding for granulocyte macrophage colony-stimulating factor [ONCOS-102] | gene therapies | EMA | 2013-06-19 | — | Targovax Oy |
Yttrium(90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 | antibodies | FDA | 2012-12-03 | — | OncoTherapy Science, Inc. |
Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 | antibodies | EMA | 2012-05-25 | — | Laboratoires OncoTherapy Science France, S.A.R.L |
eribulin mesylate [Halaven] | small molecules | FDA | 2012-05-14 | 2016-01-28 | Eisai Inc. |
(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate | small molecules | FDA | 2012-03-09 | — | Threshold Pharmaceuticals, Inc. |
Evofosfamide [TH-302] | small molecules | EMA | 2012-03-05 | — | Merck KGaA |
Aldoxorubicin | small molecules | FDA | 2011-06-29 | — | Gemini Therapeutics, Inc. |
Humanized monoclonal antibody to TumorEndothelial Marker-1 | antibodies | FDA | 2011-04-29 | — | Eisai Inc. |
Ombrabulin | small molecules | EMA | 2011-04-15 | — | Sanofi-Aventis Groupe |
crenolanib | small molecules | FDA | 2011-03-18 | — | AROG Pharmaceuticals, LLC |
ombrabulin; N-{2-methoxy-5-[(Z)-2-(3,4,5-trimethozyphenyl)vinyl]phenyl}-L-serinamide hydrochloride | small molecules | FDA | 2011-03-03 | — | Sanofi-Aventis U.S., Inc. |
angiotensin 1-7 | peptides | FDA | 2010-01-29 | — | W. Jeffrey Petty, MD |
pazopanib [Votrient] | small molecules | FDA | 2009-10-20 | 2012-04-26 | Novartis Pharmaceuticals Corp |
Tivantinib [ARQ 197] | small molecules | EMA | 2009-10-08 | — | [INACTIVE] Covance Pharma Consulting Limited |
PALIFOSFAMIDE-TRIS [Zymafos] | small molecules | EMA | 2008-12-03 | — | [INACTIVE] Ziopharm Oncology Limited |
Mx-dnG1 | small molecules | FDA | 2008-06-24 | — | Epeius Biotechnologies Corporation |
Palifosfamide | small molecules | FDA | 2008-05-05 | — | ZIOPHARM Oncology, Inc. |
Fenretinide | small molecules | EMA | 2007-01-30 | — | [INACTIVE] Cancer Research UK |
Liposomal doxorubicin hydrochloride | small molecules | FDA | 2006-12-27 | — | GP-Pharm SA |
Doxorubicin hydrochloride (liposomal) | small molecules | EMA | 2006-10-27 | — | GP-Pharm S.A. |
Brostallicin | small molecules | EMA | 2005-12-23 | — | [INACTIVE] Nuvisan Oncology |
Ridaforolimus [Jenzyl] | small molecules | EMA | 2005-08-26 | — | Organon Pharma (UK) Limited |
ridaforolimus | small molecules | FDA | 2005-08-12 | — | Merck Sharp & Dohme Corp. |
trabectedin [Yondelis] | small molecules | FDA | 2004-09-30 | 2015-10-23 | Janssen Research & Development, LLC |
N-acetylsarcosyl-glycyl-L-valyl-D-allo-isoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-propyl-N-ethylamide | peptides | EMA | 2003-12-12 | — | [INACTIVE] Abbott International European Office |
N-acetyl-sarcosyl-glycyl-L-valyl-D-alloisoleucyl-L-threonyl-L-norvalyl-L-isoleucyl-L-arginyl_L-prolylethylaminde acetate | peptides | FDA | 2003-12-09 | — | AbbVie, Inc. |
digitoxin | antibodies | FDA | 2001-10-18 | — | SimRx Advisors LLC |
Trabectedin [Yondelis] | small molecules | EMA | 2001-05-30 | — | Pharma Mar S.A. |
CT-2584 Mesylate | small molecules | FDA | 1999-04-16 | — | Cell Therapeutics, Inc. |
Idoxuridine | small molecules | FDA | 1996-04-08 | — | NeoPharm, Inc. |