A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2021

Abstract

The second year of the COVID-19 pandemic had no adverse effect on the number of new drug approvals by the US Food and Drug Administration (FDA). Quite the contrary, with a total of 50 new drugs, 2021 belongs to the most successful FDA years. We assign these new drugs to one of three levels of innovation: (1) first drug against a condition (“first-in-indication”), (2) first drug using a novel molecular mechanism (“first-in-class”), and (3) “next-in-class”, i.e., a drug using an already exploited molecular mechanism. We identify 21 first-in-class, 28 next-in-class, and only one first-in-indication drugs. By treatment area, the largest group is once again cancer drugs, many of which target specific genetic alterations. Every second drug approved in 2021 targets an orphan disease, half of them being cancers. Small molecules continue to dominate new drug approvals, followed by antibodies and non-antibody biopharmaceuticals. In 2021, the FDA continued to approve drugs without strong evidence of clinical effects, best exemplified by the aducanumab controversy.

A genome sequencing system for universal newborn screening, diagnosis, and precision medicine for severe genetic diseases

“…These methods will allow the number of NBS-rWGS disorders to grow with time as further effective interventions are developed and approved. 3 The initial variants evaluated by in silico NBS-rWGS were all 29,865 rare (gnomAD allele frequency < 0.5%), germline, pathogenic (P), or likely pathogenic (LP) ClinVar nucleotide (nt) variants that mapped to the 388 NBS-rWGS disease-gene dyads. These included variants where the associated condition was not specified.…”

“…[7][8][9][10][11][12][13][14][15][16][17][18][19][20] We evaluated the analytic performance of NBS-rWGS retrospectively in 2,208 critically ill children with any suspected genetic disorder and 2,168 of their parents, who had received Dx-rWGS. [1][2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20] We queried their genomes in TileDB with the feedback loop-informed subset of ClinVar P and LP variants that mapped to the 388 NBS-rWGS disease-gene dyads (Figure 3B). Dx-rWGS reported 119 diagnoses, of which 20 where RUSP NBS disorders.…”

“…In an era of rapid impending growth in cell and gene therapies and orphan drugs, NBS-rWGS will enable conditions with newly approved, highly effective interventions to be screened without delay. 3 We anticipate that 1,000 genetic disorders may meet criteria for NBS by 2030. 3 Unlike panel tests with fixed content, NBS-rWGS conditions can be added or removed dynamically on the basis of individual, regional, or societal preferences.…”

“…1 While NBS can greatly improve health outcomes, the number of genetic disorders screened has not kept pace with genomic or therapeutic innovation. [2][3][4][5] Between 2006 and 2022, the number of core disorders that were recommended for NBS of dried blood spots (DBSs) in the United States-the Recommended Uniform Screening Panel (RUSP)-increased from 27 to 35, and the number of affected infants identified increased from 6,439 to 6,466. 4,5 However, there are 7,200 known genetic diseases and hundreds of targeted treatments that have been approved or are in clinical trials.…”

“…4,5 However, there are 7,200 known genetic diseases and hundreds of targeted treatments that have been approved or are in clinical trials. 3,6 Over the past decade, rapid whole-genome sequencing (rWGS) has developed into an effective diagnostic test (Dx-rWGS) for almost all heritable diseases and is gaining acceptance as a first-tier test for critically ill newborns with suspected genetic diseases. [7][8][9][10][11][12][13][14][15][16][17][18][19][20] rWGS is attractive for comprehensive NBS because it concomitantly examines almost all genetic diseases with similar time to result as biochemical NBS of DBSs by mass spectrometry (NBS-MS).…”