Table 1.
Selected FDA approvals 2022
| Drug name | Generic name | Indication | Molecule |
|---|---|---|---|
| Adstiladrin | Nadofaragene firadenovec-vncg | Bladder cancer | Adenovirus-5 vector-based gene therapy, containing an interferon-α2b transgene with Syn3, an excipient to improve transfection |
| Carvykti | Ciltacabtagene autoleucel | Multiple myeloma | CAR-T created ex vivo using a lentiviral vector to deliver two BCMA-targeting scFvs, a 4-1BB co-stimulatory domain and a CD3ζ signaling cytoplasmic domain |
| Daxxify | DaxibotulinumtoxinA-lanm | Wrinkles | Purified botulinum toxin type A molecule with TransMTS peptide |
| Elaherea | Mirvetuximab soravtansine-gynx | Ovarian cancer | Antibody–drug conjugate consisting of the cytotoxic maytansinoid DM4 covalently linked to the humanized mAb M9346A, which binds folate receptor 1 |
| Enjaymo | Sutimlimab-jome | Autoimmune hemolytic anemia | Humanized IgG4 mAb with subnanomolar affinity for total complement component 1 |
| Hemgenix | Etranacogene dezaparvovec-drlb | Hemophilia | AAV5 vector with the FIX-Padua mutant |
| Imjudo | Tremelimumab-actl | Hepatocellular cancer, NSCLC | CTLA-4-blocking human IgG2 mAb |
| Kimmtrak | Tebentafusp-tebn | Uveal melanoma | Bispecific fusion protein comprising a high-affinity TCR specific to a peptide from the gp100 antigen, fused to an anti-CD3 single-chain antibody fragment |
| Lunsumio | Mosunetuzumab-axgb | Follicular lymphoma | Full-length bispecific antibody that targets CD20 on B cells and CD3 on T cells. |
| Opdualag | Nivolumab relatlimab-rmbw | Melanoma | Fixed dose combination of two checkpoint inhibitor IgG4ҡ mAbs: relatlimab, which targets LAG-3, and nivolumab, an approved mAb that targets PD-1 |
| Rolvedon (Rolontis) | Eflapegrastim-xnst | Neutropenia, leukopenia | Long-acting peptide G-CSF receptor agonist produced by covalent coupling of a human G-CSF analog (18.6 kDa) and an Fc fragment of human IgG4 (49.8 kDa) via a 3.4-kDa polyethylene glycol linker |
| Skyrizi | Risankizumab-rzaa | Crohn’s disease | Humanized IgG1 mAb that selectively binds p19 subunit of human IL-23 cytokine and inhibits its interaction with IL-23 receptor |
| Skysona | Elivaldogene autotemcel | Adrenoleukodystrophy | Autologous CD34+ hematopoietic stem cells transduced with lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. |
| Spevigo | Spesolimab-sbzo | Psoriasis | Humanized IgG1 anti-IL-36 receptor mAb |
| Spikevax | COVID-19 vaccine, mRNA | COVID-19 | mRNA vaccine against coronavirus SARS-CoV-2 encoding a pre-fusion stabilized form of the spike protein |
| Tecvaylia | Teclistamab-cqyv | Multiple myeloma | Bispecific BCMA-directed CD3 T cell engager; a humanized immunoglobulin G4-PAA antibody |
| Tzield | Teplizumab-mzwv | Type 2 diabetes | Humanized, non-Fc-receptor-binding mAb against the CD3ε chain expressed on mature T lymphocytes |
| Vabysmo | Faricimab-svoa | Wet age-related macular degeneration | Humanized bispecific IgG1 mAb that binds VEGF-A on one arm and angiopoietin-2 on the other |
| Xenpozyme | Olipudase alfa-rpcp | Acid sphingomyelinase deficiency | Recombinant human acid sphingomyelinase |
| Zynteglo | Betibeglogene autotemcel | β-thalassemia | Autologous CD34+ cells transduced with human β-(A-T87Q)-globin gene under control of β-globin enhancer and locus control region |
| Notable new molecular entities | |||
| Terlivaz | Terlipressin | Hepatorenal syndrome | 1-Triglycyl-8-lysine-vasopressin, a 12-amino-acid peptide |
| Amvuttra | Vutrisiran | hATTR amyloidosis | Double-stranded 2′-fluoro,2′-methoxy-modified phosphorothioate siRNA conjugated to GalNAc |
| Vtama | Tapinarof | Psoriasis | Aryl hydrocarbon receptor agonist |
| Mounjaro | Tirzepatide | Diabetes mellitus type 2 | Peptide receptor agonist of GIP and GLP-1; a 39-amino-acid modified peptide based on the GIP sequence, containing the non-canonical amino acid aminoisobutyric acid in positions 2 and 13, a C-terminal amide, and a lysine at position 20 that is attached to 1,20-eicosanedioic acid via a linker |
aAccelerated Approval. CAR-T, chimeric antigen receptor T cell; scFv, single-chain variable fragment; BCMA, B cell maturation antigen, mAb, monoclonal antibody; IgG, immunoglobulin G; PAA, proline, alanine, alanine; mAb, monoclonal antibody; CTLA-4, cytotoxic T lymphocyte-associated antigen 4; TCR, T cell receptor; G-CSF, granulocyte colony-stimulating factor; Fc, immunoglobulin constant fragment; IL, interleukin; LAG-3, lymphocyte activating gene 3; ABCD1, ATP binding cassette subfamily D member 1; VEGF-A, vascular endothelial growth factor-A; Ang-2, angiopoietin-2; hATTR, hereditary transthyretin-mediated; GalNAc, N-acetylgalactosamine; GIP, glucose-dependent insulinotropic polypeptide; GLP-1, glucagon-like peptide-1.