First-in-class medication explained

A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition.[1] While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medications and reports on them annually, first-in-class is not considered a regulatory category. Although many first-in-class medications qualify as breakthrough therapies, Regenerative Medicine Advanced Therapies and/or orphan drugs, first-in-class status itself has no regulatory effect.

Examples

!Drug!Class!Targeted conditions!Year approved(FDA)!Year approved(EMA)
Inotuzumab ozogamicin(Besponsa)Anti-CD22 monoclonal antibody-drug conjugateRelapsed or refractory B cell precursor acute lymphoblastic leukemia[2] 2017
Tagraxofusp(Elzonris)Interleukin 3-diphtheria toxin fusion protein targeting plasmacytoid dendritic cellsBlastic plasmacytoid dendritic cell neoplasm20182021
Midostaurin(Rydapt)Multi-target tyrosine kinase inhibitor not inhibited by the D816V cKit mutationSystemic mastocytosis, myelodysplastic syndrome, acute myeloid leukemia[3] 20172017[4]
Teprotumumab(Tepezza)Anti-IGF-1R monoclonal antibodyGraves' ophthalmopathy[5] 2020
Romosozumab(Evenity)Anti-sclerostin monoclonal antibodyOsteoporosis[6] 20192019
Ocrelizumab(Ocrevus)Anti-CD20 monoclonal antibodyMultiple sclerosis[7] 20172018
Ivosidenib(Tibsovo)Small molecule inhibitor of isocitrate dehydrogenase 1Acute myeloid leukemia, cholangiocarcinoma[8] 20182023
Bempedoic acid(Nexletol)Adenosine triphosphate-citrate lyase inhibitorHypercholesterolemia[9] 20202020
Tafamidis(Vyndaqel, Vyndamax)Transthyretin chaperone (stabilizer)Familial amyloid polyneuropathy and other transthyretin amyloidoses[10] 20112019
Voxelotor(Oxbryta)Hemoglobin oxygen affinity modulatorSickle cell disease[11] 2019
Lonafarnib(Zokinvy)Farnesyltransferase inhibitorHutchinson-Gilford progeria syndrome[12] 20202022
Dupilumab(Dupixent)Interleukin-4 receptor alpha subunit inhibitorAsthma, atopic dermatitis, allergic diseases[13] 2017
Lasmiditan(Reyvow)Selective 5-HT1F serotonin receptor agonistMigraine[14] 20192022
Tazemetostat(Tazverik)Selective EZH2 inhibitorEpithelioid sarcoma[15] 2020
Tirzepatide(Mounjaro)GLP-1 and GIP activatoradult type 2 diabetes[16] 20222022

Controversy

Safety

By definition, a first-in-class drug does not have the safety evidence from analogous products that not-first-in-class drugs would have. However, a study investigating recalls and warnings in relation to first-in-class drugs approved between 1997 and 2012 by Health Canada has found that first-in-class drugs actually have a more favourable benefit-to-harm ratio.[17]

Economics

First-in-class drugs are often seen as commercially more attractive as they may tap into a market segment that has hitherto been underserved, but this may be illusory.[18] In fact, most blockbuster drugs (drugs with annual sales revenues exceeding) were not first-in-class drugs. The economic potential of a first-in-class drug, which is typically priced higher than later drugs in the same class, has been largely declining due to efforts by health insurers to restrict what specialty drugs are covered and prevent incumbency advantages.[19]

Costs

A lower number of available therapeutic options correlates with higher prices.[20] In addition, many first-in-class medications are specialty drugs and orphan drugs,[21] which means that manufacturers have to recoup development costs from a smaller market.[22] This raises ethical questions about the sustainability of the high prices on these costs.[23] [24]

References

  1. Lanthier. Michael. Miller. Kathleen L.. Nardinelli. Clark. Woodcock. Janet. 2013-08-01. An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987–2011. Health Affairs. 32. 8. 1433–1439. 10.1377/hlthaff.2012.0541. 23918488. 0278-2715.
  2. Web site: BESPONSA 1 mg powder for concentrate for solution for infusion - Summary of Product Characteristics (SmPC) - (emc). 2021-07-01. www.medicines.org.uk.
  3. Web site: Rydapt: Uses, Dosage, Side Effects, Warnings. 2021-07-01. Drugs.com. en.
  4. Web site: Rydapt European Medicines Agency . 2024-01-16 . www.ema.europa.eu.
  5. Web site: Office of the Commissioner. 2020-03-24. FDA approves first treatment for thyroid eye disease. 2021-07-01. FDA. en.
  6. Center for Drug Evaluation and Research. 2020-01-24. New Drug Therapy Approvals 2019. FDA. en.
  7. Web site: Ocrevus (ocrelizumab) Injection. 2021-07-01. www.accessdata.fda.gov.
  8. Web site: Search Orphan Drug Designations and Approvals. 2021-07-01. www.accessdata.fda.gov.
  9. Web site: DailyMed - NEXLETOL- bempedoic acid tablet, film coated. 2021-07-01. dailymed.nlm.nih.gov.
  10. Web site: Tafamidis Monograph for Professionals. 2021-07-01. Drugs.com. en.
  11. Web site: DailyMed - OXBRYTA- voxelotor tablet, film coated. 2021-07-01. dailymed.nlm.nih.gov.
  12. Web site: DailyMed - ZOKINVY- lonafarnib capsule. 2021-07-01. dailymed.nlm.nih.gov.
  13. Web site: DailyMed - DUPIXENT- dupilumab injection, solution. 2021-07-01. dailymed.nlm.nih.gov.
  14. Web site: DailyMed - Search Results for Lasmiditan. 2021-07-01. dailymed.nlm.nih.gov.
  15. Web site: Tazemetostat Monograph for Professionals. 2021-07-01. Drugs.com. en.
  16. Web site: FDA Approves Novel, Dual-Targeted Treatment for Type 2 Diabetes. 2022-06-30. fda.gov. 13 May 2022 . en.
  17. Lexchin. Joel. November 2016. How Safe and Innovative Are First-in-Class Drugs Approved by Health Canada: A Cohort Study. Healthcare Policy. 12. 2. 65–75. 1715-6572. 5221712. 28032825.
  18. Schulze. Ulrik. Ringel. Michael. 2013-06-01. What matters most in commercial success: first-in-class or best-in-class?. Nature Reviews Drug Discovery. en. 12. 6. 419–420. 10.1038/nrd4035. 23722339. 32258945. 1474-1784.
  19. Web site: Longman. Roger. 20 July 2015. The Shrinking Value of Best-in-Class and First-in-Class Drugs. 1 July 2021. In Vivo by Informa Pharma Intelligence.
  20. Kwa. Michael C.. Tegtmeyer. Kyle. Welty. Leah J.. Raney. Sam G.. Luke. Markham C.. Xu. Shuai. Kong. Betty. 2020-10-01. The relationship between the number of available therapeutic options and government payer (medicare part D) spending on topical drug products. Archives of Dermatological Research. en. 312. 8. 559–565. 10.1007/s00403-020-02042-9. 32055932. 211111984. 1432-069X.
  21. Chambers. James D.. Thorat. Teja. Wilkinson. Colby L.. Neumann. Peter J.. 2017-08-01. Drugs Cleared Through The FDA's Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process. Health Affairs. 36. 8. 1408–1415. 10.1377/hlthaff.2016.1541. 28784733. 0278-2715. free.
  22. Balancing Lower U.S. Prescription Drug Prices And Innovation – Part 1 Health Affairs Blog. Health Affairs Forefront. 2020 . en. 10.1377/forefront.20201123.804451 . Lieberman . Steven M. . Ginsburg . Paul B. . Patel . Kavita K. .
  23. Web site: Herper. Matthew. 2019-12-23. The debate over America's drug-pricing system is built on myths. 2021-07-01. STAT. en-US.
  24. Greene. Jan. January 2017. EpiPen Controversy Reveals Complexity Behind Drug Price Tags. Annals of Emergency Medicine. English. 69. 1. A16–A19. 10.1016/j.annemergmed.2016.10.025. 0196-0644. free.

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