Table 1.
Summary of 2021 FDA-approved TIDES.
| # | Active Ingredient (Trade Name) |
Indication | Therapeutic Target | Administration Route |
|---|---|---|---|---|
| Oligonucleotides | ||||
| 1 | Inclisiran (LeqvioTM) | Treatment of hypercholesterolemia | PCSK mRNA | Subcutaneously |
| 2 | Casimersen (Amondys 45) | Duchenne muscular dystrophy (DMD) amenable for exon 45 skipping | Exon 45 | Intravenously |
| Peptides | ||||
| 3 | Vosoritide (Voxzogo™) |
Achondroplasia | Natriuretic peptide receptor B (NPR-B) | Subcutaneously |
| 4 | Melphalan flufenamide (PepaxtoTM) |
Treatment of multiple myeloma (MM) and amyloid light-chain amyloidosis | Exerts anti-tumor activity through crosslinking of DNA | Intravenously |
| 5 | Voclosporin (Lupkynis™) |
Treatment of lupus nephritis | T-cells | Orally |
| 6 | Pegcetacoplan (Empaveli™) |
Treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult patients | Complement protein C3 and its activation C3b | Subcutaneously |
| 7 | Dasiglucagon (ZegalogueTM) | Hypoglycemia in diabetes patients aged over 6 years | Glucagon-receptor | Subcutaneously |
| 8 | Piflufolastat-F18 (PylarifyTM) | Positron emission tomography (PET) of prostate-specific membrane antigen (PSMA)-positive lesions in men with prostate cancer | PSMA | Intravenously |
| 9 | Difelikefalin (KorsuvaTM) | Pruritus associated with chronic kidney disease (CKD-aP) in adults undergoing hemodialysis (HD) | Kappa opioid receptor | Intravenously |
| 10 | Odevixibat (BylvayTM) | Pruritus in patients aged over 3 months with progressive familial intrahepatic cholestasis (PFIC) | Ileal bile acid transporter (IBAT) | Orally |
| Peptides in ADCs | ||||
| 11 | Tisotumab vedotin-tftv (TIVDAK™) (MMAE as cytotoxic and Valine-Citrulline as linker) |
Treatment of recurrent or metastatic cervical cancer with disease progression during or after chemotherapy | Tissue factor (TF-011), | Intravenously |
| 12 | Loncastuximab tesirine-lpyl (ZynlontaTM) (Valine-Alanine as linker) |
Treatment of adults with relapsed or refractory large B-cell lymphoma | CD19 | Intravenously |