DOI QR코드

DOI QR Code

Nomenclature of emerging therapeutics in neurology

  • Shin, Jin-Hong (Department of Neurology, Pusan National University Yangsan Hospital) ;
  • Park, Young-Eun (Department of Neurology, Pusan National University Hospital) ;
  • Kim, Dae-Seong (Department of Neurology, Pusan National University Yangsan Hospital)
  • 투고 : 2021.02.08
  • 심사 : 2021.02.25
  • 발행 : 2021.04.30

초록

New therapeutics in neurology are expanding at an unprecedented pace. In addition to the classic enzyme-replacement therapies, monoclonal antibodies are increasingly being used to modulate autoimmunity. RNA therapeutics are an emerging class, together with gene and cell therapies. The nomenclature of international nonproprietary names helps us to recognize these new drugs according to their class and function. Suffixes denote major categories of the drug, while infixes provide additional information such as the source and target.

키워드

참고문헌

  1. Robertson JS, Chui WK, Genazzani AA, Malan SF, Lopez de la Rica Manjavacas A, Mignot G, et al. The INN global nomenclature of biological medicines: a continuous challenge. Biologicals 2019;60:15-23. https://doi.org/10.1016/j.biologicals.2019.05.006
  2. Jerome JB, Sagon P. The USAN nomenclature system. JAMA 1975;232:294-299. https://doi.org/10.1001/jama.1975.03250030048023
  3. Leader B, Baca QJ, Golan DE. Protein therapeutics: a summary and pharmacological classification. Nat Rev Drug Discov 2008;7:21-39. https://doi.org/10.1038/nrd2399
  4. Mohammadi E, Seyedhosseini-Ghaheh H, Mahnam K, Jahanian-Najafabadi A, Mir Mohammad Sadeghi H. Reteplase: structure, function, and production. Adv Biomed Res 2019;8:19. https://doi.org/10.4103/abr.abr_169_18
  5. Blom D, Speijer D, Linthorst GE, Donker-Koopman WG, Strijland A, Aerts JM. Recombinant enzyme therapy for Fabry disease: absence of editing of human alpha-galactosidase A mRNA. Am J Hum Genet 2003;72:23-31. https://doi.org/10.1086/345309
  6. Babaesfahani A, Bajaj T. Glatiramer. Treasure Island: StatPearls Publishing, 2020.
  7. Mayrhofer P, Kunert R. Nomenclature of humanized mAbs: early concepts, current challenges and future perspectives. Hum Antibodies 2019;27:37-51. https://doi.org/10.3233/HAB-180347
  8. Grillo-Lopez AJ, White CA, Varns C, Shen D, Wei A, McClure A, et al. Overview of the clinical development of rituximab: first monoclonal antibody approved for the treatment of lymphoma. Semin Oncol 1999;26(5 Suppl 14):66-73.
  9. Frenzel A, Schirrmann T, Hust M. Phage display-derived human antibodies in clinical development and therapy. MAbs 2016;8:1177-1194. https://doi.org/10.1080/19420862.2016.1212149
  10. Raffaelli B, Mussetto V, Israel H, Neeb L, Reuter U. Erenumab and galcanezumab in chronic migraine prevention: effects after treatment termination. J Headache Pain 2019;20:66. https://doi.org/10.1186/s10194-019-1018-8
  11. Wang F, Zuroske T, Watts JK. RNA therapeutics on the rise. Nat Rev Drug Discov 2020;19:441-442. https://doi.org/10.1038/d41573-020-00078-0
  12. Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, et al. Comparison of long-term ambulatory function in patients with Duchenne muscular dystrophy treated with eteplirsen and matched natural history controls. J Neuromuscul Dis 2021 Feb 19. [Epub]. DOI:10.3233/JND-200548.
  13. Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, et al. Safety, tolerability, and efficacy of viltolarsen in boys with Duchenne muscular dystrophy amenable to Exon 53 skipping: a phase 2 randomized clinical trial. JAMA Neurol 2020;77:982-991. https://doi.org/10.1001/jamaneurol.2020.1264
  14. Anwar S, Yokota T. Golodirsen for Duchenne muscular dystrophy. Drugs Today (Barc) 2020;56:491-504. https://doi.org/10.1358/dot.2020.56.8.3159186
  15. Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, et al. Nusinersen versus Sham control in later-onset spinal muscular atrophy. N Engl J Med 2018;378:625-635. https://doi.org/10.1056/NEJMoa1710504
  16. Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med 2018;379:22-31. https://doi.org/10.1056/NEJMoa1716793
  17. Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 2018;379:11-21. https://doi.org/10.1056/NEJMoa1716153
  18. Keeler AM, Flotte TR. Recombinant adeno-associated virus gene therapy in light of luxturna (and Zolgensma and Glybera): where are we, and how did we get here? Annu Rev Virol 2019;6:601-621. https://doi.org/10.1146/annurev-virology-092818-015530
  19. Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med 2017;377:1713-1722. https://doi.org/10.1056/NEJMoa1706198
  20. Oh KW, Moon C, Kim HY, Oh SI, Park J, Lee JH, et al. Phase I trial of repeated intrathecal autologous bone marrow-derived mesenchymal stromal cells in amyotrophic lateral sclerosis. Stem Cells Transl Med 2015;4:590-597. https://doi.org/10.5966/sctm.2014-0212
  21. Roskoski R Jr. Properties of FDA-approved small molecule protein kinase inhibitors: a 2021 update. Pharmacol Res 2021;165:105463. https://doi.org/10.1016/j.phrs.2021.105463
  22. Campagne O, Yeo KK, Fangusaro J, Stewart CF. Clinical pharmacokinetics and pharmacodynamics of selumetinib. Clin Pharmacokinet 2021;60:283-303. https://doi.org/10.1007/s40262-020-00967-y