FDA Approves Four New Oncology/Hematology Indications in September

  • New PD-1 Inhibitor for Cutaneous Squamous Cell Carcinoma. The United States Food and Drug Administration (FDA) approved a novel PD-1 inhibitor, cemiplimab-rwlc (Libtayo®, Regeneron Pharmaceuticals), for the treatment of patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or radiation. Approval was based on results from the phase II EMOPOWER-CSCC-1 trial, as well as two expansion cohorts from a phase I trial. Among 108 patients receiving cemiplimab-rwlc in the two trials, the overall response rate (ORR) was 47%, including a 4% rate of complete response (CR). Responses lasted 6 months or longer in 61% of responding patients. There was no difference in overall response rate (ORR) between patients with locally advanced versus metastatic disease, though CR occurred more frequently in patients with metastatic disease (5% vs 0%). The most common adverse effects were fatigue, rash, and diarrhea.
  • Dacomitinib Gets First-Line Approval in NSCLC. The second-generation epidermal growth factor receptor (EGFR) targeting tyrosine kinase inhibitor (TKI) dacomitinib (Vizimpro®, Pfizer) was approved for use as a first-line treatment in patients with metastatic, non-small cell lung cancer (NSCLC) with EGFR exon 19 deletion or exon 21 L858R substitution (detected by FDA approved test). This decision was based on results from the phase III ARCHER 1050 trial, which compared dacomitinib to gefitinib in 452 patients with previously untreated metastatic NSCLC. Compared to gefitinib, dacomitinib resulted in a 5.5-month improvement in progression-free survival (PFS) (14.7 months vs 9.2 months; HR 0.59, P<.0001) and a 7.3-month improvement in OS (34.1 months vs 26.8 months; HR 0.76, P = .0438). Dacomitinib was associated with higher rates of toxicity than gefitinib, including more grade 3 rash, diarrhea, and paronychia.
  • Two Approvals for Duvelisib in CLL and Follicular Lymphoma. The phosphoinositide 3-kinase (PI3K) inhibitor duvelisib (Copiktra™, Verstem Oncology) received full approval for use in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior therapies. This approval was granted based on the phase III DUO trial, in which duvelisib was associated with a 16.4-month PFS, compared to 9.1 months with ofatumumab (HR 0.40). Additionally, duvelisib was granted accelerated approval for use in patients with relapsed or refractory follicular lymphoma who have received at least two prior systemic therapies. Accelerated approval was based on a 42% ORR in the phase II DYNAMO trial. Full approval in follicular lymphoma is contingent upon a confirmatory phase III trial. Duvelisib’s label will carry a warning regarding risk of toxicity, including infection, diarrhea or colitis, cutaneous reactions, and pneumonitis.
  • CD22-Targeted Therapy for Hairy Cell Leukemia. Moxetumomab pasudotox-tdfk (Lumoxiti™, Astra Zeneca), a CD22-targeted cytotoxic therapy, received approval for use in patients with relapsed or refractory hairy cell leukemia who have progressed on at least two prior systemic therapies, including a purine nucleoside analog. This approval was granted based on the single-arm phase III 1053 trial (N = 80), in which moxetumomab pasudotox-tdfk resulted in an ORR of 75%, including a 30% rate of durable CR. Moxetumomab pasudotox-tdfk had previously received Orphan Drug designation for this indication. The most common grade 3 or 4 adverse reactions were hypertension, febrile neutropenia, and hemolytic uremic syndrome.


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