Drugs & Therapies
VALIDATED DATAThe SMA Research Platform tracks drugs across four relevance tiers — from officially approved SMA therapies to drugs approved for other conditions where computational or clinical evidence suggests SMA relevance. Every non-SMA-approved drug has a documented reason for inclusion.
Three FDA/EMA-approved treatments target the SMN pathway directly: nusinersen (antisense oligonucleotide, intrathecal), risdiplam (small-molecule splicing modifier, oral), and onasemnogene abeparvovec (AAV9 gene therapy, one-time IV). Pipeline drugs explore complementary approaches including muscle-enhancing (apitegromab, anti-myostatin), neuroprotective (fasudil, ROCK inhibition; riluzole, glutamate modulation), HDAC-mediated SMN2 upregulation (vorinostat), NMJ strengthening (pyridostigmine, amifampridine, argx-119, nmd-670), and oxidative-stress correction (dimethyl fumarate, omaveloxolone). Each drug entry links to DiffDock virtual screening results where available.
Approved for other indications
approved other2Tracked for scientific completeness (controls, chemoinformatics); no known SMA link.
| Name | Brand | Type | Status | Mechanism | SMA relevance | |
|---|---|---|---|---|---|---|
| enasidenib | — | small_molecule | approved | — | FDA-approved IDH2 inhibitor for AML (Idhifa). Not SMA. Part of Bryzant IDH1 cancer platform. | detailinfo |
| ivosidenib | — | small_molecule | approved | — | FDA-approved IDH1 inhibitor for AML and cholangiocarcinoma (Tibsovo). Not SMA. Part of Bryzant IDH1 cancer platform. | detailinfo |
Needs review
needs review14Auto-classification fallback — awaiting human review.
| Name | Brand | Type | Status | Mechanism | SMA relevance | |
|---|---|---|---|---|---|---|
| Cisplatin | — | — | approved | Standard 1st-line chemo for CCA (with gemcitabine). | — | detailinfo |
| Dabrafenib | — | — | approved | For BRAF V600E (with trametinib). Tumor-agnostic approval. | — | detailinfo |
| Durvalumab | — | — | approved | TOPAZ-1: added to GemCis improves OS in biliary cancer. | — | detailinfo |
| Enasidenib | — | — | approved | FDA-approved for IDH2-mutant AML. Potential for IDH2-escape in CCA. | — | detailinfo |
| FOLFOX | — | — | approved | 2nd-line chemo for CCA. ABC-06 trial. | — | detailinfo |
| Futibatinib | — | — | approved | For FGFR2 fusion/rearrangement iCCA only. | — | detailinfo |
| Gemcitabine | — | — | approved | Standard 1st-line chemo for CCA (with cisplatin). | — | detailinfo |
| Ivosidenib | — | — | approved | FDA/EMA-approved for IDH1-mutant CCA. Oral, 500mg daily. ClarIDHy trial: mPFS 2.7 vs 1.4mo. | — | detailinfo |
| Pemigatinib | — | — | approved | For FGFR2 fusion/rearrangement iCCA only. | — | detailinfo |
| Trametinib | — | — | approved | For BRAF V600E (with dabrafenib). Tumor-agnostic approval. | — | detailinfo |
| Trastuzumab Deruxtecan | — | — | approved | For HER2+ solid tumors. HERB trial in biliary cancer. | — | detailinfo |
| Vorasidenib | — | — | phase3 | Dual IDH1/2 inhibitor. Approved for glioma, investigating in CCA. | — | detailinfo |
| IDH305 | — | — | phase1 | Novartis brain-penetrant IDH1 inhibitor. | — | detailinfo |
| LY3410738 | — | — | phase1 | Eli Lilly covalent dual IDH1/2 inhibitor. Overcomes D279N resistance. ORR 42.1%. | — | detailinfo |