2020 Small Molecule Drug Approvals

On Jun. 23^rd 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.

List of 2020 Small Molecule Drug Approvals

1. Zeposia (ozanimod) – oral S1P(1/5) receptor modulator – NCT02294058; NCT02047734 
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 – NCT04280705; NCT04292899; NCT04292730
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 – NCT01568073; NCT01227655 
12. Koselugo (selumetinib) –  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 – NCT02913482; NCT02908685 
16. Byfavo (remimazolam) – IV GABAA receptor modulator – NCT02290873; NCT02296892; NCT02532647 
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 – NCT02608450; NCT02608476 
26. Klisyri (tirbanibulin) – topical microtubule inhibitor – NCT03285477; NCT03285490 
27. Nexletol (bempedoic acid)  – oral ATP citrate synthase enzyme inhibitor –  NCT02666664; NCT02991118 
28. Xeglyze (abametapir) – topical metalloproteinase inhibitor – NCT02060903 ; NCT02062060 
29. Tauvid (flortaucipir F18) – IV tau protein aggregate radiodiagnostic  – NCT02516046; NCT03901092 
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 – NCT02919111; NCT02918357 
33. Artesunate (artesunate) – IV artemisinin-based antimalarial
34. Lampit (nifurtimox) – oral antiprotozoal – NCT02625974
35. Pizensy (lactitol)  – oral osmotic laxative – NCT02819297; NCT02481947 
36. Dojolvi (triheptanoin) – oral fatty acid replacement
37. Imcivree (Setmelanotide) –  subcutaneous MC4 receptor agonist – NCT02896192; NCT03287960 
38. Barhemsys (amisulpride) – IV selective dopamine D2/3 receptor antag. – NCT01991860; NCT02337062 

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.