January 2025 FDA Brief

The FDA granted priority review to sunvozertinib for the treatment of advanced NSCLC with EGFR exon 20 mutations, based on promising efficacy and safety data from the WU-KONG1 trial.

Abenacianine, a tumor-targeted fluorescent imaging agent, received fast track designation from the FDA after demonstrating its safety and efficacy in visualizing lung tumors in a phase 2 study.

The FDA’s breakthrough therapy designation for GSK’227 underscores its potential to address the critical unmet needs of patients with relapsed/refractory osteosarcoma.

The FDA cleared the investigational new drug application for REC-4539, allowing for the start of a phase 1/2 trial in small cell lung cancer and other solid tumor indications.

The FDA granted orphan drug designation to amezalpat in hepatocellular carcinoma based on phase 1b/2 data showing efficacy and safety with atezolizumab and bevacizumab in frontline treatment.

Invikafusp alfa has gained FDA fast track designation for treating TMB-H metastatic colorectal cancer.

A draft guidance from the FDA outlines criteria for confirmatory trials in accelerated approvals, emphasizing timely initiation, sponsor engagement, and new mandates for oncology treatments.

TYRA-300 has gained an investigational new drug application from the FDA and will be further studied in the phase 2 SURF302 trial for patients with low-grade, intermediate-risk non–muscle-invasive bladder cancer.

R289 has gained orphan drug status from the FDA for the treatment of patients with myelodysplastic syndromes.

The investigational new drug application for the nanoparticle drug SNB-101 has been cleared for small cell lung cancer treatment.

The FDA has granted fast track designation to emiltatug ledadotin for advanced or metastatic breast cancer treatment, as supported by new phase 1 data.

BBO-8520, an investigational oral agent, has been granted fast track designation from the FDA in previously treated KRAS G12C–mutated metastatic non–small cell lung cancer.

The phase 2 trial showed a 42% response rate with lete-cel in synovial sarcoma and MRCLS, with manageable adverse events.

LYT-200 now has gained fast track designation from the FDA in acute myeloid leukemia in addition to its prior designation for recurrent or metastatic head and neck squamous cell carcinoma.

R-5780, an oral immuno-oncology drug designed to activate gut-regulated immune pathways, will now be evaluated in a phase 1 trial for cancer treatment.

The FDA granted priority review to datopotamab deruxtecan in advanced EGFR-mutated NSCLC after prior therapies, with a decision expected by July 12, 2025.

The FDA set August 29, 2025, as the PDUFA date for the 3-month leuprolide mesylate formulation, supported by phase 3 trial data.

An FDA application for TAR-200 to treat BCG-unresponsive high-risk non-muscle-invasive bladder cancer is under real-time oncology review, expediting access to this potential treatment.

The FDA has approved the investigational new drug application of 225Ac-SSO110, allowing for the start of a phase 1/2 trial in patients with extensive-stage small cell lung cancer and Merkel cell carcinoma.

Axatilimab has gained FDA approval in 9 mg and 22 mg vials, expanding treatment options for patients with chronic GVHD after 2 prior therapy failures.

The FDA has issued a complete response letter to the biologics license application seeking the approval of tabelecleucel for Epstein-Barr virus–positive post-transplant lymphoproliferative disease treatment.

Acalabrutinib with bendamustine and rituximab is now an FDA-approved treatment for patients with previously untreated mantle cell lymphoma.

The approval of this combination is supported by data from the phase 3 CodeBreaK 300 study.

FoundationOne CDx is now FDA-approved as the first companion diagnostic for tovorafenib, enabling targeted treatment for relapsed/refractory pediatric low-grade glioma with BRAF mutations or rearrangements.

As supported by data from the phase 3 TROPION-Breast01 trial, datopotamab deruxtecan is now an FDA-approved treatment for patients with HR-positive, HER2-negative breast cancer.

LOAd703, an oncolytic adenovirus with transgenes for 4-1BBL and TMZ-CD40L, has received FDA fast track designation for pancreatic cancer treatment.

The biologics license application for RP1 (vusolimogene oderparepvec) plus nivolumab in advanced melanoma was accepted by the FDA with priority review.

Pending phase 3 trial results, a biologics license application will be submitted for the KRAS-targeting cancer vaccine ELI-002.

The FDA has granted breakthrough therapy designation to the DCISionRT test, a tool that assesses radiation therapy benefit for women aged 30 to 85 with ductal carcinoma in situ.

The combination of treosulfan and fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in acute myeloid leukemia or myelodysplastic syndromes has been approved by the FDA.