2021 Small Molecule Drug Approvals Review

We’ll be reviewing 2021’s novel FDA drug approvals in a series of articles, starting with this high-level overview of the small molecule drugs approved by the FDA in 2021. You can find Part 2 on First-in-Class Small Molecules here and the 2021 Large Molecules series here. Our deep dive on Small Molecule Oncology Approvals is also live.

together with Jen Huen, Vinicius Texeira, Naveed Yasin, Maria Koyioni, and Adrian Parodi

There were:

  • 50 novel drugs approved in 2021
  • 32 small molecules in this group (including peptides, excluding radiologics)
  • 16 large molecules
  • 27 “first-in-class” drugs

For small molecules, there were:

  • 9 in oncology
  • 5 in neurology
  • 5 for genetic conditions
  • 4 for infectious diseases
  • 3 in immunology
  • 2 in endocrinology
  • 1 in dermatology
  • 1 in hematology
  • 1 in cardiology
  • 1 in family medicine

Three of our favorite molecules are shown below:

Drug Hunter sotorasib Lumakras KRAS GTPase inhibitor atogepant Qulipta structure GCRP inhibitor ibrexafungerp structure brexafemme b-1,3-glucan synthesis inhibitor

Our complete, hi-res compilation of all 32 of the small molecule drugs with structures, drug targets and mechanisms of action, doses and routes of administration, and indication appears below. You can find past examples of our 2020 Small Molecule Approvals Poster and 2019 Small Molecule Approvals Poster online.

2021 FDA Novel Small Molecule 
Oncology
umbralisib (Ukoniq) structure
melphalan fluefenamide (Pepaxto)
sotorasib (lumakras)
infigratinib (Trueseltiq)
tivozanib (Fotivda)
mobocertinib (exkivity)
trilacicib (Cosela)
asciminib (Scemblix)
Neurology
samidorphan + alanzapine (Lybalvi)
atogepant (qulipta)
serdexmethylaphenidate + dexmethylphenidate (Azstarys)
panesimod (Ponvory)
Genetic Conditions 
maralixibat (Livmarli)
belzutifan (Welireg)
fisdenopterin (Nulibry)
vosoritide (Voxzogo)
odevixibat (Bylval)
Infectious Diseases
cabotegravir + rilpivirine (Cabenuva)
maribavir (Livtencity)
ibrexafungerp (Brexafemme)
fexinidazole (Fexinidazole)
Endocrinology
finerenone (Kerendia)
dasiglucagon (Zegalogue)
Immunology
voclosporin (Lupkynis)
avacopan (Tavneos)
belumosudil (Rezurock)
Other
difelikefalin (Korsuva)
vericiguat (Verquvo)
pegcetacoplan (Empaveli)

List of 2021 Novel Small Molecule Drug Approvals

Highlights from 2021 Small Molecule Approvals

  • Ibrezafungerp (Brexafemme) – β-1,3-glucan synthesis inhibitor
  • Atogepant (Qulipta) – calcitonin gene-related peptide
  • Sotorasib (Lumakras) – KRASG12C GTPase inhibitor

List of Novel 2021 Small Molecule Drug Approvals in Oncology (9)

  • Umbralisib (Ukoniq) – PI3Kδ and CK1ε kinase inhibitor
  • Melphalan flufenamide (Pepaxto*) – peptide-conjugated alkylating agent, withdrawn
  • Sotorasib (Lumakras) – KRASG12C GTPase inhibitor
  • Infigratinib (Truseltiq) – pan-FGFR kinase inhibitor
  • Teponitib (Tepmetko) – MET kinase inhibitor
  • Tivozanib (Fotivda) – pan-VEGFR kinase inhibitor
  • mobocertinib (Exkinity) – EGFR exon 20 insertion kinase
  • Trilaciclib (Cosela) – CDK4 and 6 kinase inhibitor
  • asciminib (Scemblix) – ABL/BCR-ABL1 kinase allosteric inhibitor

List of Novel 2021 Small Molecule Drug Approvals in Neurology (5)

  • samidorphan + olanzapine (Lybalvi) – opioid antagonist
  • atogepant (Qulipta) – calcitonin gene-related peptide antagonist
  • ponesimod (Ponvory) – sphingosine 1-phosphate (S1P) modulator
  • viloxazine (Qelbree) – selective norepinephrine reuptake inhibitor
  • serdexmethylphenidate + dexmethylphenidate (Azstarys) – catecholamine reuptake inhibitor

List of Novel 2021 Small Molecule Drug Approvals for Genetic Conditions (5)

  • maralixibat (Livmarli) – ileal bile acid transporter
  • belzutifan (Welireg) – hypoxia-inducible factor 2α
  • odevixibat (Bylvay) – ileal bile acid transporter
  • fosdenopterin (Nulibry) – cyclic pyranopterin monophosphate (cPMP)
  • vosoritide (Voxzogo) – C-type natriuretic peptide analog

List of Novel 2021 Small Molecule Drug Approvals for Infectious Diseases (4)

  • cabotegravir + rilpivirine (Cabenuva) – HIV1 integrase strand transfer
  • maribavir (Livtencity) – CMV pUL97 kinase inhibitor
  • ibrexafungerp (Brexafemme) – β-1,3-glucan sysnthesis inhibitor
  • fexinidazole (Fexinidazole) – nitroimidazole antimicrobial

List of Novel 2021 Small Molecule Drug Approvals in Endocrinology (2)

  • finerenone (Kerendia) – non-steroidal mineralocorticoid
  • dasiglucagon (Zegalogue) – antihypoglycemic, glucagon

List of Novel 2021 Small Molecule Drug Approvals in Immunology (3)

  • voclosporin (Lupkynis) – calcineurin inhibitor
  • avacopan (Tavneos) – complement C5a receptor antagonist
  • belumosudil (Rezurock) – selective ROCK2 kinase inhibitor

Other Areas (4)

  • difelikefalin (Korsuva) – kappa opioid receptor agonist
  • vericiguat (Verquvo) – guanylate cyclase stimulator
  • pegcetacoplan (Empaveli) – complement C3 inhibitor
  • estretrol + drospirenone (Nextstellis) – naturally-occurring estrogen E4

Found this helpful? Explore drughunter.com for more drug discovery resources that help you save time, stay up-to-date, and discover ideas.

You can find Part 2 on First-in-Class Small Molecules here and the 2021 Large Molecules series here. Our deep dive on Small Molecule Oncology Approvals is also live.

Navigate the full 2021 Small Molecule Drug Approvals series:

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