The Utilization of the Accelerated Approval Pathway in Oncology: A Case Study of Pembrolizumab

U.S. Food & Drug Administration. Accelerated Approval 2023 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval.

U.S. Food & Drug Administration. The history of FDA's role in preventing the spread of HIV/AIDS 2019 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/about-fda/fda-history-exhibits/history-fdas-role-preventing-spread-hivaids.

U.S. Food and Drug Administration. Guidance for industry: Expedited programs for serious conditions—drugs and biologics 2014 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/media/86377/download.

Subbiah V, Wirth LJ, Kurzrock R, et al. Accelerated approvals hit the target in precision oncology. Nat Med. 2022;28(10):1976–9.

Article  CAS  PubMed  Google Scholar 

Wong W, Kowal S, To TM, et al. The value of the accelerated approval pathway: real-world outcomes associated with reducing the time between innovations. Future Oncol. 2024;20(16):1099–110.

Article  PubMed  Google Scholar 

Gyawali B, Kesselheim AS. Reinforcing the social compromise of accelerated approval. Nat Rev Clin Oncol. 2018;15(10):596–7.

Article  PubMed  Google Scholar 

Wayant C, Mohyuddin GR, Prasad V. The accelerated approval pathway in oncology: balancing the benefits and potential harms. J Cancer Policy. 2022;32: 100323.

Article  PubMed  Google Scholar 

U.S. Food & Drug Administration. Oncology Drugs Advisory Committee (meetings from 2021-2024) 2024 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/advisory-committees/human-drug-advisory-committees/oncologic-drugs-advisory-committee.

Mehta GU, de Claro RA, Pazdur R. Accelerated approval is not conditional approval: insights from international expedited approval programs. JAMA Oncol. 2022;8(3):335–6.

Article  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval Letter (BLA 125514/0) 2014 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2014/125514Orig1s000ltr.pdf.

U.S. Food & Drug Administration. Summary Review (BLA 125514/0) 2014 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2014/125514Orig1s000SumR.pdf.

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-59 through S-64, S-69, S-76 through S-83) 2020 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2020/125514Orig1s059,s060,s061,s062,s063,s064,s069,s076,s077,s078,s079,s080,s081,s082,s083ltr.pdf.

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-14) 2017 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2017/125514Orig1s014ltr.pdf.

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-71 & S-90) 2020 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2020/125514Orig1s071,s090ltr.pdf.

Bradford D, Demko S, Jin S, et al. FDA accelerated approval of pembrolizumab for recurrent locally advanced or metastatic merkel cell carcinoma. Oncologist. 2020;25(7):e1077–82.

Article  PubMed  PubMed Central  Google Scholar 

U.S. Food & Drug Administration. Prescribing information (KEYTRUDA) 8/2021 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125514s117,s118lbl.pdf.

Geoerger B, Kang HJ, Yalon-Oren M, et al. Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1–2 trial. Lancet Oncol. 2020;21(1):121–33.

Article  CAS  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-136) 2023 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/125514Orig1s136ltr.pdf.

Friends of Cancer Research. Accelerating investigation of new therapies in earlier metastatic treatment settings 2022 [Available from: https://friendsofcancerresearch.org/wp-content/uploads/Accelerating_Investigation_Therapies_Earlier_Metastatic_Treatment_Settings.pdf.

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-16) 2017 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2017/125514Orig1s016ltr.pdf.

Langer CJ, Gadgeel SM, Borghaei H, et al. Carboplatin and pemetrexed with or without pembrolizumab for advanced, non-squamous non-small-cell lung cancer: a randomised, phase 2 cohort of the open-label KEYNOTE-021 study. Lancet Oncol. 2016;17(11):1497–508.

Article  CAS  PubMed  PubMed Central  Google Scholar 

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-088) 2020 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2020/125514Orig1s088ltr.pdf.

Cortes J, Cescon DW, Rugo HS, et al. Pembrolizumab plus chemotherapy versus placebo plus chemotherapy for previously untreated locally recurrent inoperable or metastatic triple-negative breast cancer (KEYNOTE-355): a randomised, placebo-controlled, double-blind, phase 3 clinical trial. Lancet. 2020;396(10265):1817–28.

Article  PubMed  Google Scholar 

U.S. Food & Drug Administration. Review Memo (BLA 125514/S59-64, 69, 76-83) 2020 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125514s59-64,69,76-83SumR.pdf.

The ASCO Post Staff. Breakthrough therapy designation for lambrolizumab for the treatment of advanced melanoma 2013 [Available from: https://ascopost.com/issues/may-15-2013/breakthrough-therapy-designation-for-lambrolizumab-for-the-treatment-of-advanced-melanoma/.

Kear S. FDA grants breakthrough therapy designation to pembrolizumab for NSCLC: OncLive; 2014 [Available from: https://www.onclive.com/view/fda-grants-breakthrough-therapy-designation-to-pembrolizumab-for-nsclc.

Robert C, Ribas A, Wolchok JD, et al. Anti-programmed-death-receptor-1 treatment with pembrolizumab in ipilimumab-refractory advanced melanoma: a randomised dose-comparison cohort of a phase 1 trial. Lancet. 2014;384(9948):1109–17.

Article  CAS  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-5) 2015 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2015/125514Orig1s005ltr.pdf.

Garon EB, Rizvi NA, Hui R, et al. Pembrolizumab for the treatment of non-small-cell lung cancer. N Engl J Med. 2015;372(21):2018–28.

Article  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-034) 2018 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2018/125514Orig1s034ltr.pdf.

U.S. Food & Drug Administration. Summary Review (BLA 125514/S-034) 2023 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/125514Orig1s034.pdf.

Frenel JS, Le Tourneau C, O’Neil B, et al. Safety and efficacy of pembrolizumab in advanced, programmed death ligand 1-positive cervical cancer: results from the phase Ib KEYNOTE-028 trial. J Clin Oncol. 2017;35(36):4035–41.

Article  CAS  PubMed  Google Scholar 

Chung HC, Ros W, Delord JP, et al. Efficacy and safety of pembrolizumab in previously treated advanced cervical cancer: results from the phase II KEYNOTE-158 study. J Clin Oncol. 2019;37(17):1470–8.

Article  CAS  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-53) 2019 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2019/125514Orig1s053ltr.pdf.

Chung HC, Piha-Paul SA, Lopez-Martin J, et al. Pembrolizumab after two or more lines of previous therapy in patients with recurrent or metastatic SCLC: results from the KEYNOTE-028 and KEYNOTE-158 studies. J Thorac Oncol. 2020;15(4):618–27.

Article  CAS  PubMed  Google Scholar 

U.S. Food & Drug Administration. Summary review (BLA 125514/S-14) 2019 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/125514Orig1s014.pdf.

Marcus L, Fashoyin-Aje LA, Donoghue M, et al. FDA approval summary: pembrolizumab for the treatment of tumor mutational burden-high solid tumors. Clin Cancer Res. 2021;27(17):4685–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-097) 2021 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2021/125514Orig1s097ltr.pdf.

Janjigian YY, Kawazoe A, Yanez P, et al. The KEYNOTE-811 trial of dual PD-1 and HER2 blockade in HER2-positive gastric cancer. Nature. 2021;600(7890):727–30.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Fashoyin-Aje LA, Mehta GU, Beaver JA, Pazdur R. The on- and off-ramps of oncology accelerated approval. N Engl J Med. 2022;387(16):1439–42.

Article  PubMed  Google Scholar 

U.S. Department of Health and Human Services, U.S. Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics Guidance for Industry 2023 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/media/166431/download.

U.S. Food & Drug Administration. FDA amends pembrolizumab’s gastric cancer indication 2023 [Cited 2 Jul 2024]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-amends-pembrolizumabs-gastric-cancer-indication.

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-065) 2019 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2019/125514Orig1s065ltr.pdf.

U.S. Food & Drug Administration. Summary Review (BLA 125514/S-065) 2023 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/125514Orig1s065.pdf.

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-035) 2018 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2018/125514Orig1s035ltr.pdf.

Gandhi L, Rodriguez-Abreu D, Gadgeel S, et al. Pembrolizumab plus chemotherapy in metastatic non-small-cell lung cancer. N Engl J Med. 2018;378(22):2078–92.

Article  CAS  PubMed  Google Scholar 

U.S. Food & Drug Administration. Approval letter (BLA 125514/S-8 and BLA 125514/S-12) 2016 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2016/125514Orig1s008,125514Orig1s012ltr.pdf.

Herbst RS, Baas P, Kim DW, et al. Pembrolizumab versus docetaxel for previously treated, PD-L1-positive, advanced non-small-cell lung cancer (KEYNOTE-010): a randomised controlled trial. Lancet. 2016;387(10027):1540–50.

Article  CAS  PubMed  Google Scholar 

Makker V, Taylor MH, Aghajanian C, et al. Lenvatinib plus pembrolizumab in patients with advanced endometrial cancer. J Clin Oncol. 2020;38(26):2981–92.

Article  PubMed  PubMed Central  Google Scholar 

U.S. Food & Drug Administration. Approval Letter (BLA 125514/S-105) 2021 [Cited 2 Jul 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2021/125514Orig1s105ltr.pdf.

Makker V, Colombo N, Casado Herraez A, et al. Lenvatinib plus pembrolizumab for advanced endometrial cancer. N Engl J Med. 2022;386(5):437–48.

Article  CAS  PubMed 

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