The year that brought impressive initial results from one molecular therapy, in the first systemic CRISPR trial — Intellia Therapeutics’ transthyretin amyloidosis Cas9 mRNA therapy — closed with the approval of another: Novartis’s Leqvio, the first small interfering RNA (siRNA) therapy approved for a mass market. Controversy over the pricing of bluebird bio’s Lenti-D — a lentiviral human ABCD1 cDNA gene therapy for adrenoleukodystrophy — prompted the company to withdrew its application (recalling gene therapy’s first commercial bust, uniQure’s Glybera). Two efficacy trials, from Galmed Pharmaceuticals and Inventiva Pharma, reported positive data in non-alcoholic steatohepatitis. Humanigen presented positive results for its anti-COVID-19 monoclonal antibody (mAb) lenzilumab, but the emergence of the Omicron (B.1.1.529) SARS-CoV-2 variant has exposed the vagaries of antiviral development, severely compromising the neutralizing activity of many Emergency Use Authorized COVID-19 mAbs. In January, the FDA pulled the authorizations for Lilly’s and Regeneron’s mAb cocktails. Only Vir Biotechnology’s Xevudy (sotrovimab) and the combination of amubarvimab and romlusevimab from TSB Therapeutics (Beijing) retain potency in vitro. Up next for approval are bluebird bio’s Zynteglo lentiviral gene therapy and Immunocore’s novel monoclonal T cell receptor therapy tebentafusp.
Drug/company | Indication | Drug information |
|---|
Rethymic (RVT802)/Enzyvant Therapeutics | Congenital athymia | 10/8/2021 FDA approves this partially T-cell-depleted cultured allogeneic decapsulated postnatal thymic tissue |
Tavneos (avacopan)/ChemoCentryx | ACNA-associated vasculitis | 10/8/2021 FDA approves this small- molecule inhibitor of complement factor 5a receptor |
Scemblix (asciminib)/Novartis | Chronic myelogenous leukemia | 10/29/2021 FDA grants accelerated approval to this small-molecule allosteric BCR-ABL inhibitor that selectively targets the ABL myristoyl pocket |
Susvimo (ranibizumab)/Roche | Wet age-related macular degeneration | 10/22/2021 FDA approves this antigen-binding segment of an anti-IgG1 mAb against VEGF |
Besremi (ropeginterferon alfa-2b)/PharmaEssentia | Polycythemia vera | 11/12/2021 FDA approves this single pegylated proline interferon-α2b |
Voxzogo (vosoritide)/BioMarin | Achondroplasia | 11/19/2021 FDA approves this stabilized analog of C-type natriuretic peptide containing 17 extra amino acids (PGQEHPNARKYKGANKK) appended to the native hormone’s N terminus |
Livtencity (maribavir)/Takeda | Cytomegalovirus infection | 11/23/2021 FDA approves this benzimidazole small-molecule inhibitor of UL97 kinase that interferes with viral DNA synthesis and capsid maturation |
Tezspire (tezepelumab-ekko)/Amgen | Asthma | 12/17/2021 FDA approves this fully human IgG2a mAb against thymic stromal lymphopoietin |
Vyvgart (efgartigimod alfa)/Argenx | Myasthenia gravis | 12/17/2021 FDA approves this human IgG1 antibody Fc fragment engineered with high affinity for the neonatal Fc receptor |
Leqvio (inclisiran)/Novartis | Dyslipidemia/hypercholesterolemia | 12/22/2021 FDA approves this triantennary GalNAc-conjugated phosphorothioate, 2′-O-methyl, 2′-fluoro and 2′-deoxynucleic acid–modified siRNA targeting proprotein convertase subtilisin kexin type 9 (PCSK9) mRNA |
- FDA, US Food and Drug Administration; ANCA, anti-neutrophil cytoplasmic antibodies; mAb, monoclonal antibody; GalNAc, N-acetylgalactosamine; PEG, polyethylene glycol; VEGF, vascular endothelial growth factor. Source: BioMedTracker, a service of Sagient Research (http://www.biomedtracker.com).
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