2020 Small Molecule Drug Approvals

On Jun. 23rd at 11AM PST, Dewakar Sangaraju and Dennis Hu gave a PBSS/CLSA webinar titled “Highlights from 2020’s FDA New Drug Approvals,” organized by Cyrus Khojasteh and Shichang Miao. One interesting fact about each approved small molecule drug was shared, along with statistics and specific case studies from the cohort of interest to the audience.

Subscribers can get a complete visual list with the structures of 2020 FDA new drug approvals by contacting info@drughunter.com. 2020’s large molecule FDA new drug approvals can be found here. The list of 2020 FDA approved drugs also contains drug route of administration, mechanism of action, dosing, main trial outcome or primary endpoint, NCT trial #’s for pivotal trials (Ph. III unless otherwise specified), number of patients enrolled in the pivotal trials at the time of approval, specialty and indication, all summarized from the FDA drug labels or clinical trial entries linked below. There is no meaning to the order in which drugs are presented.

Disclaimer: This information is shared for educational purposes only, is intended for research professionals, is not medical advice, is not shared on behalf of any of the contributors’ employers, and is not an endorsement of any of these products. Complete safety information can be found in each drug’s FDA label.

Zeposia (ozanimod)
Nurtec ODT (rimegepant)
Tazverik (tazemetostat)
Retevmo (selpercatinib)
Rukobia (fostemsavir)
Veklury (remdesivir) 
Orladeyo (berotralstat) 
Inqovi (decitabine and cedazuridine) 
Gemtesa (vibegron) 
Zokinvy (lonafarnib)
Ongentys (opicapone)
Koselugo (selumetini) 
Zepzelca (lurbinectedin) 
Orgovyx (relugolix) 
Evrysdi (risdiplam)
Byfavo (remimazolam) 
Olinvyk (oliceridine) 
Ayvakit (avapritinib) 
Tabrecta (capmatinib)
Gavreto (pralsetinib)
Pemazyre (pemigatinib)
Qinlock (ripretinib)
Tukysa (tucatinib) 
Isturisa (osilodrostat) 
Winlevi (clascoterone) 
Klisyri (tirbanibulin) 
Nexletol (bempedoic acid) 
Xeglyze (abametapir) 
Tauvid (flortaucipir F18) 
Detectnet (copper Cu 64 dotatate injection) 
Cerianna (fluoroestradiol F18) 
Gallium 68 (Gallium 68 PSMA-11)
Artesunate (artesunate) 
Lampit (nifurtimox)
Pizensy (lactitol) 
Dojolvi (triheptanoin)
Imcivree (Setmelanotide) 
Barhemsys (amisulpride)
oral S1P(1/5) receptor modulator
oral CGRP GPCR antagonist
oral EZH2 methyltransferase inhibitor
oral RET fusion kinase inhibitor (WT+mut)
oral HIV-1 gp120 attachment inhibitor
IV nucleotide RNA polymerase inhibitor
oral plasma kallikrein inhibitor
oral nucleoside cytidine deaminase inhibitor
oral selective β3 adrenergic receptor agonist
oral farnesyltransferase inhibitor
oral COMT enzyme inhibitor
oral MEK1/2 kinase inhibitor
IV DNA alkyating agent
oral GnRH hormone receptor antagonist
oral SMN RNA splicing modifier
IV GABAA receptor modulator
IV opioid receptor agonist
oral PDGFRA + KIT kinase inhibitor
oral MET kinase inhibitor
oral RET fusion kinase inhibitor (WT+mut)
oral FGFR1/2/3 kinase inhibitor
oral KIT + PDGFRA kinase inhibitor
oral HER2 kinase inhibitor
oral cortisol synthesis inhibitor
topical androgen receptor inhibitor
topical microtubule inhibitor
oral ATP citrate synthase enzyme inhibitor
topical metalloproteinase inhibitor
IV tau protein aggregate radiodiagnostic
IV somatostatin rcpt. (SSTR2) radiodiagnostic
IV ER-binding radiodiagnostic
IV PSMA-binding radiodiagnostic
IV artemisinin-based antimalarial
oral antiprotozoal
oral osmotic laxative
oral fatty acid replacement
subcutaneous MC4 receptor agonist
IV selective dopamine D2/3 receptor antag.

List of 2020 Small Molecule Drug Approvals

  1. Zeposia (ozanimod) – oral S1P(1/5) receptor modulator – NCT02294058NCT02047734 
  2. Nurtec ODT (rimegepant) – oral CGRP GPCR antagonist – NCT03461757 
  3. Tazverik (tazemetostat  – oral EZH2 methyltransferase inhibitor – NCT02601950 
  4. Retevmo (selpercatinib) – oral RET fusion kinase inhibitor (WT+mut) – NCT03157128 
  5. Rukobia (fostemsavir) –  oral HIV-1 gp120 attachment inhibitor – NCT02362503 
  6. Veklury (remdesivir) – IV nucleotide RNA polymerase inhibitor – NCT04280705NCT04292899NCT04292730
  7. Orladeyo (berotralstat) – oral plasma kallikrein inhibitor – NCT3485911 
  8. Inqovi (decitabine and cedazuridine) – oral nucleoside cytidine deaminase inhibitor – NCT02103478 NCT03306264
  9. Gemtesa (vibegron) –  oral selective β3 adrenergic receptor agonist – NCT03492281
  10. Zokinvy (lonafarnib)-  oral farnesyltransferase inhibitor – NCT00425607 ; NCT00916747 
  11. Ongentys (opicapone)- oral COMT enzyme inhibitor – NCT01568073NCT01227655 
  12. Koselugo (selumetini) –  oral MEK1/2 kinase inhibitor – NCT01362803
  13. Zepzelca (lurbinectedin) –  IV DNA alkyating agent – NCT02454972 
  14. Orgovyx (relugolix) – oral GnRH hormone receptor antagonist – NCT03085095
  15. Evrysdi (risdiplam)- oral SMN RNA splicing modifier – NCT02913482NCT02908685 
  16. Byfavo (remimazolam) – IV GABAA receptor modulator – NCT02290873NCT02296892NCT02532647 
  17. Olinvyk (oliceridine)  – IV opioid receptor agonist  – NCT02820324 
  18. Ayvakit (avapritinib)  – oral PDGFRA + KIT kinase inhibitor – NCT02508532 
  19. Tabrecta (capmatinib) – oral MET kinase inhibitor – NCT02414139 
  20. Gavreto (pralsetinib) – oral RET fusion kinase inhibitor (WT+mut) – NCT03037385 
  21. Pemazyre (pemigatinib) – oral FGFR1/2/3 kinase inhibitor – NCT02924376 
  22. Qinlock (ripretinib) – oral KIT + PDGFRA kinase inhibitor – NCT03353753 
  23. Tukysa (tucatinib) – oral HER2 kinase inhibitor – NCT02614794 
  24. Isturisa (osilodrostat) – oral cortisol synthesis inhibitor – NCT02180217 
  25. Winlevi (clascoterone) – topical androgen receptor inhibitor – NCT02608450NCT02608476 
  26. Klisyri (tirbanibulin) – topical microtubule inhibitor – NCT03285477NCT03285490 
  27. Nexletol (bempedoic acid)  – oral ATP citrate synthase enzyme inhibitor –  NCT02666664NCT02991118 
  28. Xeglyze (abametapir) – topical metalloproteinase inhibitor – NCT02060903 NCT02062060 
  29. Tauvid (flortaucipir F18) – IV tau protein aggregate radiodiagnostic  – NCT02516046NCT03901092 
  30. Detectnet (copper Cu 64 dotatate injection) – IV somatostatin rcpt. (SSTR2) radiodiagnostic – NCT04334837 
  31. Cerianna (fluoroestradiol F18)  – IV ER-binding radiodiagnostic – NCT01986569
  32. Gallium 68 (Gallium 68 PSMA-11) – IV PSMA-binding radiodiagnostic – NCT02919111NCT02918357 
  33. Artesunate (artesunate) – IV artemisinin-based antimalarial
  34. Lampit (nifurtimox) – oral antiprotozoal – NCT02625974
  35. Pizensy (lactitol)  – oral osmotic laxative – NCT02819297NCT02481947 
  36. Dojolvi (triheptanoin) – oral fatty acid replacement
  37. Imcivree (Setmelanotide) –  subcutaneous MC4 receptor agonist – NCT02896192NCT03287960 
  38. Barhemsys (amisulpride) – IV selective dopamine D2/3 receptor antag. – NCT01991860NCT02337062 

List of Novel APIs Among FDA Drug Approvals in 2020 (Formatted Without Links)

  • ozanimod (Zeposia) – oral S1P1/5 receptor modulator
  • rimegepant (Nurtec ODT) – oral CGRP GPCR antagonist
  • tazemetostat (Tazverik) – oral EZH2 methyltransferase inhibitor
  • selpercatinib (Retevmo) – oral RET fusion kinase inhibitor (WT + mutant)
  • fostemvasir (Rukobia) – oral HIV-1 gp120 attachment inhibitor
  • remdesivir (Veklury) – IV nucleotide RNA polymerase inhibitor
  • berotralstat (Orladeyo) – oral plasma kallikrein inhibitor
  • cedazuridine (Inqovi) – oral nucleoside cytidine deaminase inhibitor
  • vibegron (Gemtesa) – oral selective B3 adrenergic receptor agonist
  • Ionafarnib (Zokinvy) – oral farnesyltransferase inhibitor
  • opicapone (Ongentys) – oral COMT enzyme inhibitor
  • selumetinib (Koselgo) – oral MEK1/2 kinase inhibitor
  • lurbinectedin (Zepelca) – IV DNA alkylating agent
  • relugolix (Orgovyx) – oral GnRH hormone receptor antagonist
  • risdiplam (Evrysdi) – oral SMN RNA splicing modifier
  • remimazolam (Byfavo) – IV GABAA receptor modulator
  • oliceridine (Olinvyk) – IV opioid receptor agonist
  • avapritinib (Ayvakit) – oral PDGFRA + KIT kinase inhibitor
  • capmatinib (Tabrecta) – oral MET kinase inhibitor
  • pralsetinib (Gavreto) – oral RET fusion kinase inhibitor (WT+mut)
  • pemigatinib (Pemazyre) – oral FGFR1/2/3 kinase inhibitor
  • ripretinib (Qinlock) – oral KIT + PDGFRA kinase inhibitor
  • tucatinib (Tukysa) – oral HER2 kinase inhibitor
  • osildrostat (Isturisa) – oral cortisol synthesis inhibitor
  • clascoterone (Winlevi) – topical androgen receptor inhibitor
  • tirbanibulin (Klisyri) – topical microtubule inhibitor
  • bempedoic acid (Nexletol) – oral ATP citrate synthase enzyme inhibitor
  • abametapir (Xeglyze) – topical metalloproteinase inhibitor
  • flortaucipir F18 (Tauvid) – IV tau protein aggregate radiodiagnostic
  • 64Cu dotatate (Detectnet) – IV somatostatin rcpt. (SSTR2) radiodiagnostic
  • fluoroestradiol 18F (Cerianna) – IV ER-binding radiodiagnostic
  • 68Ga PSMA-11 – IV PSMA-binding radiodiagnostic
  • artesunate (Artesunate) – IV artemisinin-based antimalarial
  • nifurtimox (Lampit) – oral antiprotozoal
  • lactitol (Pizensy) – oral osmotic laxative
  • triheptanoin (Dojolvi) – oral fatty acid replacement
  • amisulpride (Barhemsys) – IV selective dopamine D2/3 receptor antagonist
  • setmelanotide (Imcivree) – subcutaneous MC4 receptor agonist

More details will be shared after the webinar on Jun. 23rd. Explore drughunter.com for more drug discovery articles.

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