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FDA Approves 3 Treatments, Cancer Vaccine Shows Promise


The Food and Drug Administration recently approved treatment for some patients with neuroblastoma, kidney cancer and bladder cancer. Elsewhere, a cancer vaccine made headlines for the treatment of patients with melanoma.

The FDA is certainly staying busy as 2023 comes to a close, approving three treatments for patients with cancer last week alone.

Additionally, exciting study findings were released regarding a cancer vaccine for the treatment of melanoma.

FDA Approves Iwilfin for High-Risk Neuroblastoma in Adults and Children

The FDA approved has Iwilfin (eflornithine) for the treatment of adult and pediatric patients with high-risk neuroblastoma who have shown at least a partial response to a previous multiagent modality therapy, which includes anti-GD2 immunotherapy.

Notably, the FDA reported that this drug is the first approval of a therapy used to reduce the risk of relapse in children with high-risk neuroblastoma. Neuroblastoma, as the National Cancer Institute explained on its website, is a cancer of immature nerve cells that most typically occurs in children, and often begins in the adrenal glands.

The FDA said the major efficacy outcome measure behind the approval was event-free survival (EFS; time after treatment when cancer does not come back or worsen; disease progression), while other notable findings included overall survival (OS; length of time from diagnosis or start of treatment when a patient is still alive).

FDA Approves Welireg for Advanced Kidney Cancer Subtype

The FDA approved Welireg ((beluztifan) for patients with advanced renal cell carcinoma (RCC) who have been previously treated with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor, and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

The approval was based on findings from the LITESPARK-005 trial. Trial findings presented earlier this year at the European Society of Medical Oncology Annual Congress (ESMO) showed that at a median follow-up of 18.4 months, patients treated with Welireg experienced objective response rates (patients whose disease responded partially or completely to treatment) of 21.9%, compared with 3.5% among patients treated with Afinitor (everolimus), and 12-month progression-free survival (PFS; the time a patient lives without their disease spreading or worsening) rates were 33.7% on Welireg versus 17.6% with Afinitor, and 18-month PFS rates were 22.5% and 9%, respectively.

FDA Approves Padcev-Keytruda Combo in Advanced Bladder Cancer

The FDA has additionally approved Padcev (enfortumab vedotin-ejfv) plus Ketruda (pembrolizumab) for patients with locally advanced or metastatic bladder cancer. Alongside this, the FDA previously granted an accelerated approval for this patient population who are unable to be treated with cisplatin-containing chemotherapy.

In the recent EV-302/KN-A39 trial, both OS and PFS showed significant improvements among the Padcev plus Keytruda group of patients, as the median overall survival for these patients was 31.5 months, while a cohort treated with chemotherapy experienced a median OS of 16.1 months.

Regarding progression-free survival, the median was 12.5 months for the Padcev-Keytruda combination group and 6.3 months in the chemotherapy group.

Cancer Vaccine Plus Keytruda Reduces Risk of Recurrence or Death in Melanoma

Patients with advanced-stage melanoma continue to experience reduced risk of recurrence or death following treatment with a combination of mRNA vaccine mRNA-4157 (V940), an investigational individualized neoantigen therapy (INT), and Keytruda, according to recent study findings.

The mRNA-4157 (V940) and Keytruda combination reduced the risk of recurrence or death by 49% and the risk of distant metastasis or death by 62% when compared with treatment with Keytruda alone in patients with stage 3 or 4 melanoma with high risk of recurrence following complete resection, as determined by the phase 2b KEYNOTE-942/mRNA-4157-P201 study, according to a news release from drug manufacturers Moderna and Merck.

The latest findings, from a median planned follow-up of approximately three years, build on previously announced primarily analysis data from a median planned follow-up of approximately two years which showed that the combination reduced the risk of recurrence or death by 44% and the risk of distance metastasis or death by 65% when compared with Keytruda alone.

Based on the trial’s findings, the Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to the combination for the post-surgical treatment of patients with high-risk melanoma earlier this year.

Moderna and Merck have also announced the phase 3 INTerpath-001 (V940-001) clinical trial to evaluate the combination as an adjuvant treatment for patients with resected, high-risk stage 3B to 4 melanoma, which is currently enrolling participants, as well as a phase 3 trial for the treatment of patients with non-small cell lung cancer treated with the combination regimen.

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