Table 1.
Summary of FDA-approved TIDES in 2023.
| # | Active Ingredient (Trade Name) |
Indication | Therapeutic Target | Administration Route |
|---|---|---|---|---|
| Oligonucleotides | ||||
| 1 | Tofersen (QalsodyTM) | To treat amyotrophic lateral sclerosis in adults who have an SOD1 gene mutation | Superoxide dismutase 1 (SOD1) mRNA | Intrathecally |
| 2 | Avacincaptad pegol (IzervayTM) | To treat geographic atrophy secondary to age-related macular degeneration | C5 complement protein | Intravitreally |
| 3 | Nedosiran (RivflozaTM) | To lower urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function | RNA-induced silencing complex (RISC) | Subcutaneously |
| 4 | Eplontersen (WainuaTM) | To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis | Transthyretin (TTR) mRNA | Subcutaneously |
| Peptides | ||||
| 5 | Flotufolastat F-18 (PoslumaTM) | To use with positron emission tomography (PET) imaging in certain patients with prostate cancer | Prostate-specific membrane antigen (PSMA) | Intravenously |
| 6 | Motixafortide (AphexdaTM) | To use with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma | Chemokine receptor 4 (CXCR4) | Intramuscularly |
| 7 | Rezafungin (RezzayoTM) | To treat candidemia and invasive candidiasis | 1,3-β-D-glucan synthase enzyme | Intravenously |
| 8 | Trofinetide (DaybueTM) | To treat Rett syndrome | Inflammatory cytokines | Orally |
| 9 | Zilucoplan (ZilbrysqTM) | To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody-positive | C5 protein | Subcutaneously |