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Table 1 FDA approvals in 2019–2020 for precision drugs with no control arm

From: Analysis of new treatments proposed for malignant pleural mesothelioma raises concerns about the conduction of clinical trials in oncology

Drug

Trial

Number of Patients

Comments*

References

Tazemetostat

NCT02601950

62

Efficacy was investigated in a single-arm cohort (Cohort 5) of a multi-center trial (Study EZH-202, NCT02601950) in patients with histologically confirmed, metastatic or locally advanced epithelioid sarcoma.”

[8, 9]

Avapritinib

NCT02508532

43

Efficacy was investigated in NAVIGATOR (NCT02508532), a multi-center, single-arm, open-label trial enrolling 43 patients with GIST harboring a PDGFRA exon 18 mutation

[10]

Enfortumab Vedotin

NCT03219333

125

Efficacy was investigated in EV-201 (NCT03219333), a single-arm, multicenter trial enrolling 125 patients with locally advanced or metastatic urothelial cancer who received prior treatment with a PD-1 or PD-L1 inhibitor and platinum-based chemotherapy.”

[11]

Zanubrutinib

NCT03206970

NCT02343120

86

32

Efficacy was evaluated in BGB-3111-206 (NCT03206970), a phase 2 open-label, multicenter, single-arm trial of 86 patients with MCL who received at least one prior therapy. Efficacy was also assessed in BGB-3111-AU-003 (NCT 02343120), a phase 1/2, open-label, dose-escalation, global, multicenter, single-arm trial of B‑cell malignancies, including 32 previously treated MCL patients treated with zanubrutinib administered orally at 160 mg twice daily or 320 mg once daily.”

[12]

Entrectinib

ALKA

NCT02097810

NCT02568267

54

51

Efficacy in NTRK-positive tumors was investigated in 54 adult patients who received entrectinib at various doses and schedules in one of three multicenter, single-arm, clinical trials: ALKA, STARTRK-1 (NCT02097810) and STARTRK-2 (NCT02568267)

Efficacy in ROS1-positive metastatic NSCLC was investigated in 51 adult patients who received entrectinib at various doses and schedules in the same three trials; 90% received entrectinib 600 mg orally once daily.”

[13,14,15,16]

Selinexor

NCT02336815

122

“Efficacy was evaluated in 122 patients enrolled in Part 2 of STORM (KCP-330-012; NCT02336815), a multicenter, single-arm, open-label study of patients with RRMM who had previously received three or more anti-myeloma treatment regimens including an alkylating agent, glucocorticoids, bortezomib, carfilzomib, lenalidomide, pomalidomide, and an anti-CD38 monoclonal antibody… …The approval was based on efficacy and safety in a prespecified subgroup analysis of 83 patients”.

[4]

  1. *Comments Sources:
  2. 1. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-tazemetostat-advanced-epithelioid-sarcoma;
  3. 2. https://cacmap.fda.gov/drugs/resources-information-approved-drugs/fda-approves-avapritinib-gastrointestinal-stromal-tumor-rare-mutation;
  4. 3. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-enfortumab-vedotin-ejfv-metastatic-urothelial-cancer;
  5. 4. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-zanubrutinib-mantle-cell-lymphoma;
  6. 5. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-entrectinib-ntrk-solid-tumors-and-ros-1-nsclc;
  7. 6.https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-selinexor-multiple-myeloma.