A Look Back at the FDA News From October 2023

October was a busy month for the FDA and the field of oncology, as the regulatory agency granted 8 approvals for patients with metastatic colorectal cancer, KRAS G12C- and BRAF V600E-mutated locally advanced or metastatic non–small cell lung cancer, RET-mutated solid tumors, resected stage IIB or IIC melanoma, IDH1-mutated myelodysplastic syndromes, and advanced nasopharyngeal carcinoma.

The agency also granted 4 fast track designations, 3 orphan drug designations, and 3 investigational new drug applications across cancer types. While a partial clinical hold was placed on the investigational new drug application for lacutamab (IPH4102), pausing new patient enrollment in 2 trials, the agency lifted the hold placed on the phase 1 clinical trial (NCT05514717) of XMT-2056 in HER2-high or -low advanced or recurrent solid tumors.

Additionally, the FDA’s Oncologic Drug Advisory Committee (ODAC) met twice to discuss eflornithine (DFMO) in pediatric patients with high-risk neuroblastoma and sotorasib (Lumakras) in KRAS G12C-mutated locally advanced or metastatic non–small cell lung cancer.

In a 14 to 6 vote during the first meeting, they agreed that eflornithine (DFMO) showed sufficient evidence to reduce the risk of relapse in this group of patients who are in remission and have completed multi-agent, multi-modality therapy. During the second meeting, the FDA’s ODAC voted 10 to 2 that the progression-free survival of sotorasib (Lumakras) cannot be reliably interpreted in the CodeBreaK 200 (NCT04303780) trial.

Here’s a look back at the notable FDA updates from October 2023.

FDA Fast Tracks AVB-001 for Relapsed/Refractory Ovarian Cancer

On October 2, 2023, the FDA granted a fast track designation to AVB-001 as a treatment option for adult patients with relapsed/refractory ovarian cancer.

FDA and Takeda to Withdraw Mobocertinib for EGFR Exon20+ NSCLC

Also on October 2, 2023, the FDA and Takeda announced plans working towards a voluntary withdrawal of mobocertinib (Exkivity) for adult patients with EGFR exon20 insertion mutation-positive locally advanced or metastatic non–small cell lung cancer whose disease has progressed on or after platinum-based chemotherapy in the United States.

FG001 Gains FDA Orphan Drug Designation for High-Grade Glioma

The FDA granted an orphan drug designation to FG001 on October 3, 2023, as an optical imaging agent for the visualization of malignant tissue during surgery for patients with high-grade glioma.

FDA Grants FTD to BI 764532 in DLL3+ SCLC and Neuroendocrine Carcinoma

On October 3, 2023, the FDA granted a fast track designation to BI 764532 as an option for patients with extensive-stage small cell lung cancer that has progressed after at least 2 prior lines of treatment, including platinum-based chemotherapy, and for patients with advanced or metastatic extrapulmonary neuroendocrine carcinoma whose disease has progressed following at least 1 prior line of treatment, including platinum-based chemotherapy.

FDA’S ODAC Votes Yes to Eflornithine for Pediatric Neuroblastomas

In a 14-to-6 vote, the FDA’s ODAC agreed that eflornithine shows sufficient evidence to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma who are in remission and have completed multi-agent, multi-modality therapy on October 4, 2023.

FDA Approves Companion Diagnostic for Panitumumab in mCRC

The FDA granted approval to the CRCdx RAS Mutation Detection Kit on October 5, 2023, to be used as a companion diagnostic for panitumumab (Vectibix) for patients with metastatic colorectal cancer.

FDA’s ODAC Votes No for Sotorasib in KRAS G12C-Mutated NSCLC

Also on October 5, 2023, in a 10-to-2 vote, the FDA’s ODAC voted that the progression-free survival of sotorasib (Lumakras) cannot be reliably interpreted vs docetaxel in the CodeBreaK 200 (NCT04303780) study for adult patients with KRAS G12C-mutated locally advanced or metastatic non–small cell lung cancer, as determined by an FDA-approved test, who have received at least 1 prior systemic therapy.

FDA Places Clinical Hold on IND of Lacutamab for Lymphomas

On October 5, 2023, the FDA also placed a partial clinical hold on the investigational new drug application for lacutamab (IPH4102), leading to a pause in new patient enrollment in the ongoing phase 2 TELLOMAK trial (NCT03902184) and phase 1b peripheral T-cell lymphoma trial (​​NCT05321147).

FDA Accepts BLA Resubmission of OBI Presentation of Pegfilgrastim-cbqv

The biologics license application for the on-body injector presentation of pegfilgrastim-cbqv (Udenyca OnBody) was resubmitted to the FDA on October 6, 2023. The resubmission follows the FDA’s completion of its inspection of a third-party filler.

FDA Approves Companion Diagnostic for Selpercatinib in RET Fusion+ Solid Tumors

On October 10, 2023, the FDA approved the FoundationOne® CDx for use as a companion diagnostic for selpercatinib (Retevmo) for the treatment of adult patients with locally advanced or metastatic solid tumors harboring a RET gene fusion whose disease has progressed on or following prior systemic treatment, or who have no satisfactory alternative treatment options.

FDA Grants Orphan Drug Designation to SLS009 for AML

The FDA granted an orphan drug designation to SLS009 (GFH009) for the treatment of patients with acute myeloid leukemia on October 11, 2023.

FDA Greenlights Encorafenib/Binimetinib for BRAF V600E-Mutated NSCLC

The FDA approved encorafenib (Braftovi) and binimetinib (Mektovi) for patients with metastatic non–small cell lung cancer harboring a BRAF V600E mutation, as detected by an FDA-approved test, on October 11, 2023.

FDA Okays CDx for Encorafenib Plus Binimetinib in BRAF V600E-Mutated NSCLC

On October 12, 2023, the FDA granted approval to FoundationOne® CDx and FoundationOne Liquid CDx for use as companion diagnostics to aid in the identification of patients with metastatic non–small cell lung cancer that harbors a BRAF V600E mutation, and who may benefit from treatment with the combination of encorafenib (Braftovi) plus binimetinib (Mektovi).

FDA Approves Adjuvant Nivolumab in Resected Stage IIB or IIC Melanoma

The FDA approved nivolumab (Opdivo) as monotherapy on October 13, 2023, for the adjuvant treatment of patients with resected stage IIB or IIC melanoma.

FDA Clears IND of TERN-701 for Chronic Myeloid Leukemia

On October 16, 2023, the FDA cleared the investigational new drug application and the design of the phase 1 CARDINAL trial, evaluating the safety, tolerability, pharmacokinetics, and efficacy of TERN-701 in patients with chronic myeloid leukemia.

FDA Approves Perioperative Pembrolizumab Plus Chemotherapy in Early NSCLC

The FDA approved perioperative pembrolizumab (Keytruda) in combination with platinum-containing chemotherapy as neoadjuvant treatment, and then continued as a single agent in the adjuvant setting for the treatment of patients with resectable stage II, IIIA, or IIIB (T3-4N2) non–small cell lung cancer on October 16, 2023.

DOC1021 Gains FDA Fast Track Designation in Glioblastoma Multiforme

On October 17, 2023, the FDA granted a fast track designation to DOC1021, a dendritic cell vaccine, as a potential treatment option for patients with glioblastoma multiforme.

FDA Grants Orphan Drug Designation to TTX101 in Malignant Gliomas

The FDA granted an orphan drug designation to TTX101, a nanoformed drug candidate, on October 19, 2023, for potential use in patients with malignant gliomas.

FDA Approves Ivosidenib for IDH1-Mutated MDS

The FDA approved ivosidenib (Tibsovo) tablets for adult patients with relapsed/refractory myelodysplastic syndromes with a susceptible IDH1 mutation as detected by an FDA-approved test on October 24, 2023.

FDA Accepts IND Application of MB-109 for GBM and High-Grade Astrocytoma

On October 26, 2023, the FDA accepted the investigational new drug application of MB-109 for the treatment of recurrent glioblastoma and high-grade astrocytoma.

FDA Greenlights Toripalimab in Advanced Nasopharyngeal Carcinoma

The FDA granted approval to the combination of toripalimab-tpzi (Loqtorzi) with cisplatin and gemcitabine for the first-line treatment of adult patients with metastatic or recurrent locally advanced nasopharyngeal carcinoma and as a monotherapy for the treatment of adult patients with recurrent, unresectable, or metastatic nasopharyngeal carcinoma with disease progression on or after platinum-containing chemotherapy on October 27, 2023.

SLS009 Earns FDA Fast Track Designation for R/R PTCL

SLS009, a novel CDK9 inhibitor, was granted a fast track designation from the FDA for the treatment of relapsed or refractory peripheral T-cell lymphoma on October 30, 2023.

FDA Grants Permission to Proceed Studies of Roginolisib Across Cancer Types

On October 31, 2023, the FDA granted permission to proceed with clinical investigations of roginolisib (IOA-244) in the United States.

FDA Lifts Hold on Study of XMT-2056 in HER2+ Recurrent/Metastatic Solid Tumors

Also on October 31, 2023, the FDA lifted the clinical hold placed on the phase 1 clinical trial (NCT05514717) of XMT-2056, a systemically administered Immunosynthen STING-agonist antibody-drug conjugate, as monotherapy for the treatment of patients with HER2-high or -low advanced or recurrent solid tumors.