Last Month in Oncology: FDA Cancer News Roundup

Last month, the US Food and Drug Administration (FDA) approved three new drugs, as well as a potentially practice-changing cancer screening tool, and expanded indications for two agents. The FDA also placed a partial hold on a phase 3 lung cancer trial and a drug company voluntarily withdrew its bladder cancer drug. 
Here’s a snapshot of what happened in October.
New drugs
1. The FDA approved zolbetuximab (Vyloy, Astellas Pharma) to treat advanced gastric cancer in the first-line setting. Zolbetuximab — the first claudin 18.2–targeting agent approved in the United States — is indicated in combination with fluoropyrimidine and platinum-containing chemotherapy to treat locally advanced unresectable or metastatic human epidermal growth factor 2 (HER2)-negative gastric or gastroesophageal junction adenocarcinoma that is claudin 18.2 positive. Alongside the drug approval, the FDA also okayed the Ventana CLDN18 (43-14A) RxDx Assay (Ventana Medical Systems, Inc. and Roche Diagnostics) to identify claudin 18.2–positive tumors.
Approval was based on two trials, which showed a median overall survival benefit of 2.2-2.7 months in patients who received zolbetuximab plus chemotherapy vs placebo plus chemotherapy.
2. The FDA approved inavolisib (Itovebi, Genentech) in the first-line setting alongside palbociclib and fulvestrant to treat advanced breast cancer in patients with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative disease after recurrence on or after adjuvant endocrine therapy. The FDA also approved the FoundationOne Liquid CDx assay to identify patients who are eligible for inavolisib.
The approval was based on the INAVO120 trial, which found patients in the inavolisib arm demonstrated a median progression-free survival benefit about two times longer than those in the placebo group. 
3. The agency also approved Hympavzi (marstacimab, Pfizer) as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients aged ≥ 12 years who have hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. Prophylaxis with the once-weekly subcutaneous injection reduced bleeding episodes compared with both on-demand and prophylactic standard treatment with clotting factor replacement.
Expansions
1. The FDA has expanded the indication for nivolumab (Opdivo, Bristol Myers Squibb) in patients with non–small cell lung cancer (NSCLC). Nivolumab is now approved in combination with platinum-doublet chemotherapy in the neoadjuvant setting, followed by adjuvant nivolumab alone after surgery in untreated adults with resectable stages IIA-IIIB disease and no known EGFR mutations or ALK rearrangements.
2. The FDA has granted accelerated approval to asciminib (Scemblix, Novartis) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase, moving the indication to the first-line setting. Approval was based on the ASC4FIRST trial, which found a major molecular response rate of 68% among patients randomized to asciminib vs 49% among those randomized to investigators’ choice of an older tyrosine kinase inhibitor. 
Withdrawals and Clinical Holds
1. Gilead Sciences said it is voluntarily withdrawing its bladder cancer drug, sacituzumab govitecan (Trodelvy), after a confirmatory trial in patients with metastatic urothelial cancer failed to meet its primary goal of improved overall survival. The FDA consulted on the decision, which does not impact the other approved indications for the drug in advanced breast cancer.
Sacituzumab govitecan received accelerated approval to treat metastatic urothelial cancer in 2021 based on objective response and duration of response rates. In the confirmatory trial, however, more deaths occurred in the sacituzumab govitecan group — a finding Gilead had said it was investigating.
2. The FDA placed a partial clinical trial hold on the phase 3 PRESERVE-003 trial evaluating gotistobart (BioNTech and OncoC4) in NSCLC. The partial hold was placed after an independent data monitoring committee “identified a possible variance in population results,” according to a regulatory document. The partial hold will not affect patients already enrolled in the trial, who will continue to receive treatment, or other ongoing trials for gotistobart.
Screening Approvals
The FDA approved Cologuard Plus — a next-generation multitarget stool DNA test from Exact Sciences, developed in collaboration with Mayo Clinic — in adults aged ≥ 45 years at average risk for colorectal cancer. The approval was based on the results of the BLUE-C study, which found the next-generation test had sensitivities of 95% for colorectal cancer and 43% for advanced precancerous lesions, as well as a specificity of more than 90% for both.
 
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