US Food and Drug Administration (FDA) approvals in 3Q show no effect of the pandemic, with roughly the same number of approvals as in 3Q19. The agency granted three accelerated approvals for cancer indications, including two that target CD19 in unique ways (Gilead’s Tecartus, which has circulating malignant cells removed during manufacturing, and MorphoSys’s Monjuvi, which has modifications to the Fc portion). The first small-molecule exon-skipping drug, Roche’s Evrysdi, was approved. The saga of Biogen’s monoclonal aducanumab may finally come to a resolution in early 2021, when the FDA reviews data from its restarted continuation trial for Alzheimer’s disease. Both the European Medicines Agency (EMA) and FDA gave thumbs down to BioMarin’s hemophilia A drug valoctocogene roxaparvovec.
FDA approvals by drug type
Drug approvals stay the course, pandemic or no.
Notable clinical trial results (3Q20)
Drug/company | Indication | Drug information |
---|---|---|
TT11 CD30 CAR-T/Tessa Therapeutics | Hodgkin’s lymphoma | 7/23/2020 In a phase1/2 trial of CAR-T cells against CD-30 in heavily pretreated patients, 59% had complete response, 72% had overall response (J. Clin. Oncol. https://doi.org/10.1200/JCO.20.01342, 2020) |
Nemolizumab/Galderma | Atopic dermatitis | 7/9/2020 In a phase 3 double-blind placebo controlled trial of this subcutaneous human monoclonal against IL-31AR, patients receiving the drug experienced greater reduction in pruritus than those receiving placebo (N. Engl. J. Med. 383, 141–150, 2020) |
Veklury (remdesivir)/Gilead Sciences | COVID-19 | 8/21/2020 Patients randomized to a 5-day course of remdesivir had a statistically significant difference in clinical status compared with standard care (JAMA https://doi.org/10.1001/jama.2020.16349, 2020) |
Notable drug approvals (3Q20)
Drug/company | Indication | Drug information |
---|---|---|
Tecartus (brexucabtagene autoleucel)/Gilead Sciences | Mantle cell lymphoma | 7/24/2020 FDA grants accelerated approval for this CD19 chimeric antigen receptor (CAR)-T cell therapy from which circulating malignant cells are removed during manufacture |
Monjuvi (tafasitamab)/MorphoSys | Diffuse large cell lymphoma | 7/31/2020 FDA grants accelerated approval for this humanized monoclonal antibody against the B-cell receptor CD19, with a modified Fc region to enhance antibody-dependent cellular toxicity |
Blenrep (belantamab mafodotin)/GlaxoSmithKline | Multiple myeloma | 8/5/2020 FDA grants accelerated approval for this first-in-class antibody–drug conjugate, a humanized antibody directed against B cell maturation antigen (BCMA) and conjugated to auristatin F via a non-cleavable linker |
Viltepso (viltolarsen)/Nippon Shinyaku | Duchenne muscular dystrophy | 8/12/2020 FDA approves this exon-53-specific antisense morpholino oligonucleotide |
Gavreto (pralsetinib)/Blueprint Medicines | Non-small-cell lung cancer | 9/4/2020 FDA approves this small-molecule inhibitor of RET (rearranged during transfection) for patients with RET fusion mutations |
Evrysdi (risdiplam)/Roche | Spinal muscular atrophy | 8/7/2020 FDA approves this small- molecule modulator of SMN2 pre-mRNA splicing |
Olinvyk (oliceridine)/Trevena | Acute pain | 8/7/2020 FDA approves this first-in-class GPCR inhibitor |
Upcoming catalysts (1Q21)
Drug/company | Indication | Drug information |
---|---|---|
Aducanumab/Biogen | Alzheimer’s disease | 3/5/2021 FDA PDUFA date for this fully human IgG1 monoclonal against a conformational epitope on β-amyloid plaques |
Idecabtagene vicleucel/Bristol Myers Squibb | Multiple myeloma | 3/6/2021 FDA PDUFA date for this chimeric antigen receptor (CAR)-T cell targeting BCMA |
Casimersen/Sarepta Therapeutics | Duchenne muscular dystrophy | 2/25/2021 FDA PDUFA date for this phosphorodiamidate morpholino oligonucleotide that skips exon 45 of the mutated dystrophin |
Tanezumab/Pfizer | Osteoarthritis and osteoarthritis pain | 12/01/2020, 1/1/2021 FDA PDUFA and EMA CHMP (respectively) decisions on this anti-NGF IgG2Δa monoclonal antibody |
Evinacumab/Regeneron | Dyslipidemia /hypercholesterolemia | 2/11/2021 FDA PDUFA date for this fully human monoclonal antibody made by VelocImmune technology and targeting angiopoietin-like-3 (ANGLPTL3) |
Notable regulatory setbacks (3Q20)
Drug/company | Indication | Drug information |
---|---|---|
Valoctocogene roxaparvovec/ BioMarin | Hemophilia A | 8/18/2020, 9/9/2020 FDA and EMA ask for data going out to one (EMA) and two (FDA) years to show durable effect |
Jyseleca/Gilead Sciences | Rheumatoid arthritis | 8/18/2020 FDA issues a complete response letter for this small-molecule JAK1 inhibitor because of toxicity concerns and incomplete data from trial looking at effects on sperm production |
UCARTCS1/Cellectis | Multiple myeloma | 7/6/2020 FDA issues clinical hold on this CAR-T therapy targeting a cell-surface protein overexpressed in multiple myeloma due to treatment-emergent cardiac arrest |
Leronlimab/CytoDyn | HIV/AIDS | 7/13/2020 FDA issues refuse-to-file letter for this humanized IgG4 monoclonal antibody directed against CCR5 used in combination with antiviral therapy, citing not enough data for a substantive review |
PBGM01/Passage Bio | GM1 gangliosidosis | 8/13/2020 FDA puts a clinical hold on this AAVhu68 capsid-delivered GLB1 gene encoding lysosomal acid β-galactosidase due to biocompatibility of the proposed intra-cisterna magna delivery device |
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DeFrancesco, L. Drug pipeline 3Q20. Nat Biotechnol 38, 1230–1231 (2020). https://doi.org/10.1038/s41587-020-0721-y
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DOI: https://doi.org/10.1038/s41587-020-0721-y