February 2024: FDA Approvals, Fast Tracks, and Regulatory Action

The FDA had a busy February 2024, reviewing and approving a wide range of new therapies and making significant regulatory decisions.

Patients with multiple myeloma patients saw the approval of teclistamab-cqyv (Tecvayli) with a reduced dosing schedule, linvoseltamab (REGN5458), and epcoritamab-bysp (Epkinly). Liposomal irinotecan (Onivyde) received first-line treatment approval for pancreatic cancer, and IMM-1-104 and 9MW2821 gained fast track designations for their potential in treating the disease.

The combination of osimertinib (Tagrisso) with chemotherapy for EGFR-mutant lung cancer was approved by the FDA, and tepotinib (Tepmetko) received full approval for specific genetic alterations in non-small cell lung cancer. Additionally, the FDA prioritized reviewing new lung cancer treatments.

Notably, lifileucel (LN-144) became the first FDA-approved therapy for patients with advanced melanoma who have exhausted treatment options. Moreover, the FDA approved tislelizumab (Tevimbra) for gastric cancer, granted orphan drug designation for IO-202 in chronic myelomonocytic leukemia, and accepted applications for various therapies targeting other cancer types.

Here’s a look back on all the FDA happenings from the month of February 2024.

FDA Fast Tracks BST02 for Liver Cancer

The T-cell therapy BST02 was granted a fast track designation from the FDA on February 1, 2024, for the treatment of patients with all forms of liver cancer, including those with hepatocellular carcinoma and cholangiocarcinoma.

FDA Clears IND for ACE2016 in EGFR-Expressing Solid Tumors

The FDA cleared the investigational new drug application for ACE2016, an allogeneic γδ2 T-cell therapy for the treatment of patients with solid tumors that EGFR on February 4, 2024.

FDA ODAC to Review Ide-Cel Data in Multiple Myeloma

On February 5, 2024, it was announced that the FDA’s Oncologic Drug Advisory Committee (ODAC) will convene on March 15, 2024, to review data from the phase 3 KarMMa-3 study (NCT03651128) of idecabtagene vicleucel (ide-cel; Abecma) in patients with triple class-exposed relapsed/refractory multiple myeloma.

FDA Fast Tracks UV1, Ipilimumab, and Nivolumab in Malignant Mesothelioma

Also on February 5, 2024, the FDA granted a fast track designation to the combination of UV1 with ipilimumab (Yervoy) and nivolumab (Opdivo) for the treatment of patients with unresectable malignant pleural mesothelioma.

Vepdegestrant Gains FDA Fast Track Designation in ER+/HER2- Breast Cancer

The FDA granted a fast track designation to vepdegestrant as a monotherapy for the treatment of adult patients with ER-positive/HER2-negative locally advanced or metastatic breast cancer that received prior treatment with endocrine-based therapy on February 5, 2024.

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FDA Accepts Supplemental Biologics License Application of Nivolumab/Chemo in NSCLC

On February 7, 2024, the FDA accepted the supplemental biologics license application for neoadjuvant nivolumab plus chemotherapy for operable non-small cell lung cancer treatment, representing a potential new therapy option for these patients.

FDA Halts Clinical Studies of Magrolimab in AML, MDS

All studies of magrolimab (Hu5F9-G4) in hematologic malignancies, including expanded access programs, have been placed on full clinical hold by the FDA as of February 7, 2024.

Soquelitinib Receives FDA Orphan Drug Designation for T-Cell Lymphomas

On February 8, 2024, the FDA granted an orphan drug designation to soquelitinib (formerly known as CPI-818) for the treatment of patients with T-cell lymphoma.

FDA Lifts Clinical Hold on CAR T Study in AML

The FDA lifted the clinical hold on the investigational new drug application for HEMO-CAR-T, a proprietary chimeric antigen receptor T-cell therapy for the treatment of acute myeloid leukemia (AML) following the issue of a clinical hold letter on February 9, 2024.

FDA Accepts Resubmitted NDA for Dasynoc in CML and ALL

The resubmission of the new drug application for Dasynoc, an optimized version of dasatinib (Sprycel) for the treatment of chronic myeloid leukemia and acute lymphoblastic leukemia was accepted by the FDA on February 12, 2024.

FDA Grants Breakthrough Therapy Designation to Novel Agent in GEP-NETs

Also on February 12, 2024, 212PB-DOTAMTATE (AlphaMedixTM) received breakthrough therapy designation from the FDA, signifying the drug's potential to significantly improve over existing treatments for inoperable or metastatic, somatostatin receptor-expressing gastroenteropancreatic neuroendocrine tumors.

BXCL701 Gains Fast Track Designation for mCRPC Treatment

The FDA granted a fast track designation to the combination of BXCL701 with an immune checkpoint inhibitor for the treatment of patients with metastatic small cell neuroendocrine prostate cancer with progression on chemotherapy and no evidence of microsatellite instability on February 12, 2024.

FDA Approves First-Line NALIRIFOX for Metastatic PDAC

On February 13, 2024, the FDA has approved liposomal irinotecan, 5-fluorouracil, leucovorin, and oxaliplatin (NALIRIFOX) as a first-line treatment for patients with metastatic pancreatic ductal adenocarcinoma.

FDA Accepts sNDA of Repotrectinib for Solid Tumors With NTRK Alterations

The supplemental new drug application for repotrectinib (Augtyro) was accepted by the FDA on February 14, 2024, for the treatment of patients aged 12 and older with solid tumors that have an NTRK gene fusion and are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity.

Denileukin Diftitox BLA Resubmitted for CTCL After Addressing FDA Concerns

The biologics license application for denileukin diftitox (Lymphir) was resubmitted to the FDA for the treatment of patients with relapsed/refractory cutaneous T-cell lymphoma after at least 1 prior systemic therapy on February 14, 2024.

FDA Grants Full Approval to Tepotinib for Non-Small Cell Lung Cancer

As of February 15, 2024, tepotinib is an FDA-approved treatment option for patients with non-small cell lung cancer with MET exon 14 skipping alterations.

FDA Greenlights IND of KME-0584 for AML and MDS Therapy

On February 15, the FDA also cleared an investigational new drug application for KME-0584 for relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndrome treatment.

Tigilanol Tiglate Wins FDA Orphan Drug Designation for Sarcoma

Tigilanol tiglate (Stelfonta), an intratumoral drug, received an orphan drug designation from the FDA for the treatment of soft tissue sarcoma on February 16, 2024.

FDA OKs Lifileucel Treatment for Advanced Melanoma

The FDA approved lifileucel on February 16, 2024, for the treatment of patients with advanced melanoma who have progressed following anti-PD-1/L1 and/or targeted therapy, making it the first FDA-approved therapy for this patient population.

FDA Oks Osimertinib Plus Chemo in EGFR-Mutant Lung Cancer

Also on Februay 16, 2024, the FDA approved the combination of osimertinib plus chemotherapy for the treatment of locally advanced or metastatic EGFR-mutated non-small cell lung cancer.

FDA Accepts Dato-DXd Application for Non-Small Cell Lung Cancer

The biologics license application of datopotamab deruxtecan (Dato-DXd) in previously treated, advanced, nonsquamous non-small cell lung cancer was accepted by the FDA on February 19, 2024.

FDA Grants Priority Review for Vorasidenib in IDH-Mutant Diffuse Glioma

On February 20, 2024, the FDA and European Medicines Agency accepted vorasidenib (AG-881) applications, suggesting imminent approval for the treatment of IDH-mutant diffuse glioma.

Pembrolizumab/Chemo Gets FDA Priority Review in Endometrial Cancer

Also on February 20, 2024, the FDA granted priority review to the supplemental biologics license application for pembrolizumab (Keytruda) plus chemotherapy for the treatment of advanced endometrial cancer.

FDA to Review Adagrasib/Cetuximab Combo in KRAS G12C-Mutated CRC

The FDA also accepted a supplemental new drug application for priority review regarding the combination of adagrasib (Krazati) with cetuximab (Erbitux) for treating previously treated locally advanced or metastatic colorectal cancer with a KRAS G12C mutation on February 20, 2024.

FDA Approves sBLA for Reduced Teclistamab Dose in R/R Multiple Myeloma

The FDA approved the reduced dosing frequency of 1.5 mg/kg every 2 weeks for teclistamab-cqyv in patients with relapsed/refractory multiple myeloma who have sustained a complete response or better for at least 6 months, also on February 20, 2024.

FDA Fast Tracks IMM-1-104 for PDAC Treatment

On February 20, 2024, IMM-1-104, a novel drug targeting the MAPK pathway, received a fast track designation from the FDA for the treatment of pancreatic cancer after first-line therapy failure.

Linvoseltamab Earns FDA Priority Review for Multiple Myeloma

A biologics license application for linvoseltamab was accepted for priority review by the FDA on February 21, 2024, as a therapeutic option for adult patients with relapsed/refractory multiple myeloma who have progressed after 3 or more prior lines of therapy.

IO-202 Receives FDA Orphan Drug Designation in CMML

On February 21, 2024, IO-202 was granted an FDA orphan drug designation for treating chronic myelomonocytic leukemia, addressing an unmet need.

FDA Approves IND Application of IMC001 for EpCAM+ Gastrointestinal Tumors

The FDA cleared the investigational new drug application for IMC001 as a potential treatment option for patients with EpCAM-positive advanced gastrointestinal tumors on February 22, 2024.

FDA Revokes Melphalan Flufenamide Approval, Citing Efficacy/Safety Concerns

The FDA withdrew the approval of melphalan flufenamide (Pepaxto; Melflufen) on February 23, 2024, due to the confirmatory OCEAN trial (NCT03151811) not showing its clinical benefit and the lack of demonstrated safety and effectiveness.

FDA Awards Breakthrough Therapy Designation to Novel HER2-Activating NSCLC Therapy

The FDA granted a breakthrough therapy designation to BAY 2927088, an oral, small molecule tyrosine kinase inhibitor for patients with non-small cell lung cancer harboring HER2-activating mutations on February 26, 2024.

FDA Accepts Tislelizumab BLA for Advanced Gastric and GEJ Cancers

On February 27, 2024, the biologics license application of tiselizumab, an anti-PD-1 monoclonal antibody, plus chemotherapy for the first-line treatment of gastric or gastroesophageal junction cancer was accepted by the FDA.

FDA Grants Priority Review to Epcoritamab for R/R Follicular Lymphoma

The FDA has prioritized the review of the supplemental biologics license application for epcoritamab-bysp for treating adult relapsed or refractory follicular lymphoma after at least 2 lines of therapy on February 27, 2024.

FDA Fast Tracks 9MW2821 for Esophageal Squamous Cell Carcinoma

Also on February 27, 2024, the FDA granted a fast track designation to 9MW2821 as a potential treatment option for patients with advanced, recurrent, or metastatic esophageal squamous cell carcinoma.

FDA Fast Tracks IDP-023 for Non-Hodgkin Lymphoma and Myeloma

The FDA granted a fast track designation to IDP-023, a natural killer cell therapy, for the treatment of patients with non-Hodgkin lymphoma and multiple myeloma on February 29, 2024.