Approvals rolled on, with some notable firsts: the first ever FDA-approved drug for Hutchison–Gilford progeria and European approval for a small-interfering RNA (siRNA) in a blockbuster indication—Novartis/Alnylam’s cholesterol-lowering Leqvio (inclisiran), targeting proprotein convertase subtilisin kexin type 9 (PCSK9). Another siRNA drug, Oxlumo (lumasiran) for hyperoxaluria, was also registered at the FDA. Several gene and cell-based therapies ran into problems, mainly due to manufacturing issues or an inability to inspect third-party manufacturing sites. In infectious disease, registrations came for two new Ebola monoclonal antibody (mAb) treatments, one a cocktail of three recombinant molecules and the other a mAb derived from the plasma of a survivor of the 2015 outbreak; interim reporting on COVID-19 trials is ramping up, with more to come in the coming quarters.
Drug/company | Indication | Drug information |
|---|
Evinacumab/Regeneron | Dyslipidemia/hypercholesterolemia | 12/10/2020 In a double-blind, placebo-controlled, phase 2 trial, a fully human IgG4 mAb against angiopoietin-like 3 reduced low-density lipoprotein cholesterol by more than 50% at the maximum dose (N. Engl. J. Med. 383, 2307–2319, 2020) |
Nadofaragene firadenovec/FKD Therapies Oy | Bladder cancer | 11/27/2020 In a phase 3, open label, multi-dose trial, over 50% of patients receiving this adenoviral serotype 5 vector carrying an interferon-α 2b transgene with a polyamide surfactant excipient (Syn3) for enhanced transduction of the bladder epithelium had a complete response at 3 months (Lancet https://doi.org/10.1016/S1470-2045(20)30540-4) |
Imetelstat/Geron | Myelodysplastic syndrome | 10/27/2020 In this phase 2/3 trial, this 13-mer N3′–P5′ thio-phosphoramidate (NPS) oligonucleotide covalently attached to a palmitoyl lipid moiety caused durable transfusion independence in heavily transfused patients (J. Clin. Oncol. 39, 48–56, 2021) |
Lirentelimab/Allakos | Gastroenteritis | 10/22/2020 In a phase 2 randomized, placebo-controlled trial, this anti–Siglec-8 IgG1 mAb reduced gastrointestinal eosinophils (N. Engl. J. Med. 383, 1624–1634, 2020) |
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