January 2024 FDA News Signals Progress in Cancer Treatment

In January 2024, the FDA granted 3 approvals, including to pembrolizumab (Keytruda), external beam radiotherapy, and concurrent chemotherapy for cervical cancer; DermaSensor, the first AI-powered tool to diagnose skin cancer; and erdafitinib (Balversa) for advanced bladder cancer.

Several promising therapies received fast track designation (FTD), like sotorasib (Lumakras) with avutometinib (VS-6766) for KRAS-mutated lung cancer and SLS009 for relapsed/refractory acute myeloid leukemia (AML). Numerous investigational new drug (IND) applications were also approved for novel therapies across various cancers, including ovarian, glioblastoma (GBM), pancreatic, and solid tumors.

Further, manufacturing changes for axicabtagene ciloleucel (axi-cel; Yescarta) will lead to quicker availability for patients, and the FDA reopened review for the ProSense system, offering hope for patients with early-stage breast cancer.

Here is a look back at the FDA happenings from the month of January 2024.

FDA Rejects sNDA for Sotorasib in KRAS G12C-Mutated NSCLC

On January 2, 2024, the FDA rejected the supplemental new drug application (NDA) for sotorasib for patients with previously treated locally advanced or metastatic KRAS G12C-mutated non–small cell lung cancer (NSCLC).

FDA Grants Orphan Drug Designation to DOC1021 for Malignant Glioma

The FDA granted DOC1021 orphan drug designation (ODD) for malignant glioma, including GBM, on January 3, 2024.

FDA Lifts Partial Clinical Hold on Lacutamab IND

On January 4, 2023, the FDA lifted the partial clinical hold placed on the IND application of lacutamab (IPH4102). The phase 2 TELLOMAK study (NCT03902184) investigating lacutamab in patients with advanced T-cell lymphoma will proceed.

FDA Places Clinical Hold on Trial of LN-45 in NSCLC

The phase 2 IOV-LUN-202 trial (NCT04614103) investigating the LN-45 tumor infiltrating lymphocyte therapy for the treatment of patients with advanced NSCLC has been placed on clinical hold by FDA following the death of a patient on January 4, 2024. The fatal adverse event is potentially related to the non-myeloablative lymphodepletion preconditioning regimen.

FDA Grants FTD to Rinatabart Sesutecan for FRα-Expressing Ovarian Cancer

The FDA has granted a FTD to rinatabart sesutecan (PRO1184-S) for the treatment of patients with folate receptor alpha-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer.

FDA Grants Safe to Proceed Letter to IND of Ropidoxuridine in GBM

On January 8, 2024, the FDA granted a Safe to Proceed letter to the IND application of a phase 2 study evaluating ropidoxuridine (IPdR) as a radiation sensitizing agent during radiotherapy in patients with newly diagnosed IDH-wildtype GBM with unmethylated MGMT promoter status.

FDA Clears IND of NUV-1511 for Advanced Solid Tumors

Also on January 8, 2024, an IND application to evaluate NUV-1511 for the treatment of patients with solid tumors was cleared by the FDA. With this IND clearance, a phase 1/2 clinical study of NUV-1511 plans to begin.

FDA Issues Complete Response Letter to Zolbetuximab BLA in GI Cancer

On January 9, 2024, the FDA issued a complete response letter to the biologics license application (BLA) of zolbetuximab for the upfront treatment of patients with locally advanced, unresectable, or metastatic HER2-negative, Claudin 18.2-positive gastric or gastroesophageal junction adenocarcinoma.

FDA Approves sBLA for Tisotumab Vedotin in Cervical Cancer

The FDA also accepted the supplemental BLA (sBLA) to convert the accelerated approval of tisotumab vedotin-tftv (Tivdak) to full approval for patients with recurrent or metastatic cervical cancer whose disease has progressed on or after first-line therapy on January 9, 2024.

FDA Clearance of ACT-IOP-003 Launches Phase 1b Study in Pancreatic Cancer

The FDA cleared an IND application for ACT-IOP-003, an implantable iontophoretic product, as a potential treatment for patients with pancreatic cancer on January 9, 2024. A phase 1b trial of the agent plans to start mid-2024.

FDA Ok’s NDA for SH-105 in Breast and Ovarian Cancer

On January 9, 2024, the FDA also accepted an NDA of SH-105, a ready-to-dilute formula of an existing treatment for adenocarcinoma of the breast or ovary.

TME Pharma Prepares IND Filing for Olaptesed Pegol in GBM Following FDA Meeting

TME Pharma, manufacturer of olaptesed pegol, (NOX-A12), a CXCL12 inhibitor used for the treatment of GBM, completed a pre-IND advice meeting with the FDA on January 10, 2024, and plans to file its IND application and expedited regulatory pathway request by the end of Q1 2024.

FDA Grants Fast Track Designation to SLS009 in R/R AML

The FDA granted a FTD to SLS009 (formerly GFH009), a novel and highly selective CDK9 inhibitor, for the treatment of relapsed/refractory AML on January 11, 2024.

FDA Grants Fast Track Designation to RC88 in Ovarian Cancer

On January 12, 2024, the FDA granted a FTD to RC88 for the treatment of platinum-resistant ovarian, fallopian tube, and peritoneal cancers.

FDA Approves Pembrolizumab Plus Chemoradiotherapy in High-Risk Cervical Cancer

The FDA approved pembrolizumab plus external beam radiotherapy and concurrent chemotherapy as a treatment for patients with newly diagnosed, high-risk, locally advanced cervical cancer on January 12, 2024.

FDA Approves First AI-Powered Skin Cancer Diagnostic Tool

On January 17, 2024, the FDA approved DermaSensor, the first AI-powered tool to diagnose skin cancer, including melanoma, basal cell carcinoma, and squamous cell carcinoma, at the point of testing and noninvasively.

FDA Expands HT-001 Trial to Include Cancers With EGFR-Associated Toxicities

The FDA cleared the implementation of a number of protocol amendments for an ongoing trial (NCT05639933) evaluating HT-001 for the treatment of patients with cancer undergoing treatment of skin toxicities associated with EGFR inhibitors on January 18, 2024.

FDA Grants Premarket Approval to TTFields in NSCLC

The FDA accepted the premarket approval application of tumor treating fields plus standard systemic therapies on January 18, 2024, for the treatment of NSCLC.

FDA Grants Avutometinib/Sotorasib FTD in KRAS-Mutated NSCLC

The FDA fast tracked avutometinib combined with sotorasib for treating KRAS G12C-mutant NSCLC in patients who haven't previously used a KRAS G12C inhibitor and have received at least 1 prior therapy on January 19, 2024.

FDA Oks Erdafitinib for Advanced Bladder Cancer

Also on January 19, 2024, the FDA approved erdafitinib for the treatment of locally advanced or metastatic urothelial carcinoma with FGFR3 genetic alterations who have experienced disease progression on or following at least 1 prior line of treatment.

FDA Grants Orphan Drug Designation to PTX-252 in AML

The FDA has granted an ODD to PTX-252 in AML on January 22, 2024.

FDA Clears Clinical Trial of NK010 in Ovarian Cancer

The FDA has granted clearance for a clinical trial of NK010 for the treatment of specific cancer types, particularly those that are resistant to established therapies on January 22, 2024. The first indication selected for investigation in the clinical trial is ovarian cancer.

FDA Accepts BLA for Obe-Cel in R/R Acute Lymphoblastic Leukemia

The BLA for obecabtagene autoleucel (obe-cel; AUTO1) for the treatment of adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia was accepted by the FDA, also on January 22, 2024.

IND of HBM9027 for Advanced Solid Tumors Receives FDA Clearance

An IND application for HBM9027 was cleared by the FDA on January 23, 2024, allowing a first-in-human trial to evaluate the bispecific antibody in patients with advanced solid tumors to commence.

FDA Clears IND of BSI-082 for Hematologic and Solid Tumors

On January 24, 2024, the FDA granted clearance to an IND application for the novel anti-SIRPα monoclonal antibody candidate BSI-082 for the treatment of hematologic and solid tumors.

FDA Grants Priority Review to Trastuzumab in HER2+ Solid Tumors

The FDA granted priority review to the sBLA of trastuzumab deruxtecan (T-Dxd; Enhertu) for unresectable or metastatic HER2-positive solid tumors on January 29, 2024.

FDA Clears IND for OriCAR-017 in Relapsed/Refractory Multiple Myeloma

The FDA cleared an IND application for OriCAR-017 for the treatment of patients with relapsed/refractory multiple myeloma on January 29, 2024. The application is supported by findings from the POLARIS study (NCT05016778).

FDA Grants Priority Review to Liso-Cel Applications in MCL and FL

The promising chimeric antigen receptor T-cell therapy lisocabtagene maraleucel (liso-cel; Breyanzi) gained priority review for relapsed/refractory follicular lymphoma and mantle cell lymphoma in both the United States and Japan on January 29, 2024. The FDA target action dates are May 23 and May 31, 2024, respectively.

FDA Approves Manufacturing Change, Shortening Wait Time for Axi-cel

The FDA approved a manufacturing process change on January 30, 2024, resulting in a shorter manufacturing time for axi-cel, resulting in an anticipated reduced turnaround time from 16 days to 14 days.

Daratumumab Application in Myeloma Submitted to FDA

The sBLA for a daratumumab (Darzalex)-based regimen for the treatment of newly diagnosed multiple myeloma was submitted to the FDA on January 31, 2024.

FDA Reopens Review of ProSense for Early-Stage Breast Cancer Treatment

Also on January 31, 2024, and despite a previous denial, the FDA agreed to re-evaluate the ProSense System for the treatment of patients with early-stage breast cancer through a supervisory review process.

FDA Grants Priority Review to BLA of Afami-cel in Synovial Sarcoma

On January 31, 2024, the FDA has expedited its review of afamitresgene autoleucel (afami-cel), a novel engineered T-cell therapy, for treating patients with advanced synovial sarcoma.