In 2020, despite several challenges due to the COVID-19 pandemic, the FDA has approved many novel products that served previously unmet medical needs and significantly helped in advancing patient’s quality of life. The broad indication wise distribution (Figure 1) of all CDER’s 2020 drug approvals indicates notable advances in drug discovery1,2.

CDER, approved 53 novel drugs*, either as New Molecular Entities (NMEs) under New Drug Applications (NDAs: 74%) or as new therapeutic biologics under Biologics License Applications (BLAs: 26%).

New Drug Approvals (FDA) in 2020

Significant drug launches of 2020

Many of the novel entities approved in 2020 are notable for their potential positive impact and unique contributions towards medical care.

First-in-class novel drugs

40% of novel drugs (21 of 53) approved as ‘First-in-class’. Few notable approvals include:

• Rukobia (Fostemsavir, Viiv Healthcare, 07/02/2020)
  A new type of antiretroviral medication to treat HIV-1 via gp120:CD4 cellular interaction.
• Koselugo (Selumetinib, AstraZeneca LP, 04/10/2020)
  MEK1/2 (RAF-MEK-ERK) inhibitor, for treatment of certain pediatric patients with neurofibromatosis type 1 (NF1PN).

Orphan novel drugs

58% of novel drugs (31 of 53) designated as ‘Orphan status’ to treat rare diseases. Notable examples with rare diseases include:

• Evrysdi (Risdiplam, Genentech Inc, 08/07/2020)
  mRNA splicing modifier for SMN2, used as a treatment for spinal muscular atrophy (SMA).
• Lampit (Nifurtimox, Bayer Healthcare Pharms, 08/06/2020)
  The first therapy approved by FDA to treat pediatric patients with chagas disease.
• Orladeyo (Berotralstat, BioCryst Pharmaceuticals Inc, 12/03/2020)
  A plasma kallikrein inhibitor, to treat patients with hereditary angioedema (HAE).

Other notable drug approvals

• Artesunate (Amivas LLC, 05/26/2020)
  It helps in the treatment of severe malaria in adult and pediatric patients by inhibiting EXP1, a glutathione S-transferase.
• Imcivree (Setmelanotide, Rhythm Pharmaceuticals Inc, 11/25/2020) 
  It is a MC4 receptor agonist for the treatment of obesity and to control hunger associated with pro-opiomelanocortin deficiency.
• Isturisa (Osilodrostat, Novartis Pharms Corp, 03/06/2020)
  Used for adults with Cushing’s disease by blocking the enzyme known as 11-β-hydroxylase (CYP11B1) and preventing cortisol production.
• Orgovyx (Relugolix, Myovant Sciences GmbH, 12/18/2020)
  It is a GnRH receptor antagonist and is used for the treatment of certain patients with pancreatic cancer.
• Qinlock (Ripretinib, Deciphera Pharmaceuticals LLC, 05/15/2020)
  Potent pan-KIT and PDGFRα kinase inhibitor. It is the first new drug specifically approved as a fourth-line treatment for advanced gastrointestinal stromal tumor (GIST).
• Veklury (Remdesivir, Gilead Sciences Inc, 10/22/2020)
  Inhibits SARS-CoV-2 RNA-dependent RNA polymerase (RdRp). It was the first medication in the U.S. for the treatment of patients with COVID-19 infection (hospitalized adults and adolescent).
• Zokinvy (Lonafarnib, Eiger BioPharmaceuticals Inc, 11/20/2020)
  A farnesyltransferase inhibitor which is used to treat certain patients with Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies (Rare conditions caused by certain genetic mutations that leads to premature aging).

References

• US FDA