Last Month in Oncology: FDA Cancer News Roundup

Author: Pratham Sarkar

The United States Food and Drug Administration (FDA) recently issued three key regulatory decisions that illustrate shifts in the cancer treatment landscape. The agency approved four new pharmaceuticals, expanded the use of three therapies, introduced two novel formulations, declined clearance for a lung cancer drug, and oversaw the completion of trials for two investigational agents. 

One of the new approvals is cosibelimab (Unloxcyt, Checkpoint Therapeutics, Inc.), an

Credits: GlobeNewswire

immune checkpoint inhibitor for adults with metastatic or locally advanced cutaneous squamous cell carcinoma who cannot undergo curative surgery or radiation. The approval came from an open-label trial of 109 patients, showing a 47% objective response rate in metastatic cases and a 48% rate in locally advanced cases. The median duration of response was not reached for metastatic disease but was 17.7 months for locally advanced disease. 

The FDA also approved zenocutuzumab (Bizengri, Merus) for patients with non-small cell lung cancer (NSCLC) or pancreatic adenocarcinoma that has an NRG1 gene fusion. To qualify, patients must have advanced, unresectable, or metastatic disease that has progressed after prior systemic therapy. Clinical trial data revealed a 33% overall response rate in NSCLC and 40% in pancreatic cancer, with response durations ranging from 3.7 to 16.6 months. The drug has a boxed warning for embryo-fetal toxicity.

Credits: CliniExpert

Ensartinib (Ensacove, Xcovery Holdings, Inc.) was approved to treat ALK-positive, locally

advanced or metastatic NSCLC in adults who have not previously been treated with an ALK inhibitor. In the eXalt3 trial, ensartinib doubled the progression-free survival compared to crizotinib, the current first-line standard treatment.

Another new approval is remestemcel-L (Ryoncil, Mesoblast Ltd), the first allogeneic mesenchymal stromal cell therapy for children aged two months or older with steroid-refractory acute graft-vs-host disease. In the pivotal phase 3 study, 30% of patients had a complete response, and 41% had a partial response by day 28, providing an important new treatment for a condition with poor historical outcomes.

In terms of expanded use, the FDA granted a new indication for tislelizumab (Tevimbra, BeiGene, Ltd.), which is now approved in combination with chemotherapy for the first-line treatment of unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma in PD-L1–positive adults. This decision was made based on data showing a 20% reduction in the risk of death compared to chemotherapy alone.

Credits: Health and Pharma

Durvalumab (Imfinzi, AstraZeneca) became the first immunotherapy approved for limited-stage small cell lung cancer in patients whose disease has not progressed after concurrent chemotherapy and radiation. The supporting trial showed that durvalumab improved both overall survival and progression-free survival, increasing median survival to 55.9 months from 33.4 months with placebo.

The third expansion involved encorafenib (Braftovi, Array BioPharma Inc.), which is now approved in combination with cetuximab and mFOLFOX6 for BRAF V600E-mutant metastatic colorectal cancer. This decision came from data in the BREAKWATER trial, which reported a 61% objective response rate with the combination.

Credits: Oncology News Central

Alongside these therapeutic approvals, the FDA authorized two new drug formulations. A subcutaneous version of nivolumab and hyaluronidase (Opdivo Qvantig, Bristol Myers Squibb) was approved for various solid tumor indications that had been treated with intravenous nivolumab, including melanoma, lung cancer, and colorectal cancer. The CHECKMATE-67T trial showed that it was just as effective as the intravenous version and easier to administer. A new 200 mg version of pazopanib (Votrient) has also been approved, with better solubility and bioavailability, to improve treatment for advanced renal cell carcinoma and soft tissue sarcoma.

However, the FDA declined to approve a subcutaneous version of amivantamab (Rybrevant) for NSCLC due to manufacturing concerns discovered during a site inspection. These issues had nothing to do with the drug's efficacy or formulation, and the approved intravenous version is unaffected.

Finally, Merck terminated development of two investigational immunotherapies, vibostolimab and favezelimab, after trials combining them with pembrolizumab revealed insufficient benefit. This decision represents a strategic shift to focus on more promising oncology programs.

Overall, these regulatory revisions demonstrate the FDA's continuous commitment to improving cancer care while maintaining high safety and efficacy criteria.

Summary:The U.S. Food and Drug Administration (FDA) recently issued a series of important regulatory decisions that reflect evolving strategies in cancer treatment. These include approvals of four new therapies, expanded indications for three existing drugs, authorization of two novel drug formulations, the rejection of one drug application, and the discontinuation of two investigational treatments.

Among the newly approved drugs, cosibelimab (Unloxcyt) offers a new immune checkpoint inhibitor option for adults with advanced cutaneous squamous cell carcinoma who are not candidates for surgery or radiation. Zenocutuzumab (Bizengri) was approved for patients with NSCLC or pancreatic adenocarcinoma carrying NRG1 gene fusions, though it includes a boxed warning for embryo-fetal toxicity. Ensartinib (Ensacove) was approved as a first-line treatment for ALK-positive NSCLC, tripling progression-free survival over crizotinib. Remestemcel-L (Ryoncil) was the first mesenchymal stromal cell therapy licensed for children with steroid-resistant acute graft-versus-host disease. Clinical trials revealed that this therapy was effective.

The FDA also expanded the indications of three therapies: tislelizumab (Tevimbra) is now approved in combination with chemotherapy for advanced gastric and gastroesophageal cancers; durvalumab (Imfinzi) became the first immunotherapy approved for limited-stage small cell lung cancer; and encorafenib (Braftovi), in combination with cetuximab and mFOLFOX6, is now approved for BRAF V600E-mutant metastatic colorectal cancer.

Two novel medicine formulations were approved: Opdivo Qvantig, a subcutaneous form of nivolumab and hyaluronidase that provides a more practical option than intravenous dosing. Additionally, a novel 200 mg formulation of pazopanib (Votrient) that is intended to improve solubility and bioavailability was approved by the FDA.

Although the approved intravenous form of amivantamab (Rybrevant) remains available, the FDA has denied a subcutaneous version due to manufacturing issues. Merck also discontinued trials of two investigational medications, favezelimab and vibostolimab, because their interactions with pembrolizumab provided insufficient benefits.

Overall, these regulatory revisions demonstrate the FDA's continuous commitment to improving cancer care while maintaining high safety and efficacy criteria.

Works Cited

Checkpoint Therapeutics, Inc. “Checkpoint Therapeutics Announces FDA Approval of UNLOXCYT^TM (Cosibelimab-Ipdl).” GlobeNewswire News Room, Checkpoint Therapeutics, Inc, 13 Dec. 2024, www.globenewswire.com/news-release/2024/12/13/2997087/0/en/Checkpoint-Therapeutics-Announces-FDA-Approval-of-UNLOXCYT-cosibelimab-ipdl.html.

“FDA Approves Durvalumab as the First and Only Immunotherapy for LSSC Lung Cancer.” Healthandpharma.net, 5 Dec. 2024, healthandpharma.net/fda-approve-durvalumab-imfinzi-immunotherapy-lung-cancer.

“FDA Approves Ensacove (Ensartinib) for ALK-Positive Locally Advanced or Metastatic Non-Small Cell Lung Cancer-CliniExpert.” Cliniexpert.com, 15 May 2018, www.cliniexpert.com/article/1033.html.

Frellick, Marcia. “FDA Approves Subcutaneous Nivolumab for All Solid Tumor Indications.” Oncology News Central, ONC, 30 Dec. 2024, www.oncologynewscentral.com/drug/fda-approves-subcutaneous-nivolumab-for-all-solid-tumor-indications.

Stern, Victoria. “Last Month in Oncology: FDA Cancer News Roundup.” Medscape, 7 Jan. 2025, www.medscape.com/viewarticle/last-month-oncology-fda-cancer-news-roundup-2025a100009o?