August 2024 FDA Roundup: Key Approvals Drive Progress in Oncology
Here is a look back on all the FDA happenings from the month of August 2024.
In August 2024, the FDA made several significant advances in oncology, impacting a range of cancers from sarcomas to gliomas.
One of the most notable approvals was for afamitresgene autoleucel (afami-cel; Tecelra) on August 1, 2024. This accelerated approval offers a new treatment option for patients with advanced synovial sarcoma, based on data from the phase 2 SPEARHEAD-1 trial (NCT04044768). This approval was followed by the FDA's clearance of the SeCore™ CDx HLA A Sequencing System as a companion diagnostic.
Vorasidenib (Voranigo) was then approved for the treatment of patients with IDH-mutant gliomas, providing a first-in-class option. In the same field, a new drug application was submitted for TLX101-CDx, a PET imaging agent to help better characterize gliomas.
The month also saw progress in lung cancer treatment, with the FDA granting priority review to durvalumab (Imfinzi) for the potential treatment of limited-stage small cell lung cancer (SCLC). The combination of durvalumab with chemotherapy was approved for resectable non–small cell lung cancer (NSCLC), and lazertinib (Lazcluse) plus amivantamab-vmjw (Rybrevant) was approved as a first-line treatment in EGFR-mutated NSCLC.
Another critical development was the breakthrough therapy designation for fam-trastuzumab deruxtecan-nxki (T-DXd; Enhertu) in hormone receptor (HR)-positive/HER2-low breast cancer. This designation highlights the T-DXd’s potential to improve outcomes for this patient population that has limited treatment options.
Here is a look back on all the FDA happenings from the month of August 2024.
FDA Grants Afami-cel Accelerated Approval in Advanced Synovial Sarcoma
On August 1, 2024, the FDA granted an accelerated approval to afami-cel for treating patients with advanced synovial sarcoma.
Medical illustration featuring cancer cells from a microscopic to macroscopic view: © Fay Melronna - stock.adobe.com
Alemtuzumab Wins FDA Orphan Drug Designation as Part of ALL CAR T Therapy
The FDA granted orphan drug designation to alemtuzumab (CLLS52) in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) on August 1, 2024.
PGDx Elio Plasma Focus Dx Gains FDA De Novo Marketing Authorization
The FDA granted a de novo marketing authorization to the PGDx elioTM plasma focus Dx, a pan-solid tumor liquid biopsy test for tumor mutation profiling via simple blood draw, on August 2, 2024.
FDA Rejects Iomab-B BLA Application, Requests New Randomized Study
On August 5, 2024, the FDA decided that data from the SIERRA study (NCT02665065) are not sufficient to support a biologics license application filing for Iomab-B as a treatment for patients with relapsed/refractory acute myeloid leukemia.
Vorasidenib Secures FDA Approval in IDH-Mutant Diffuse Glioma
Vorasidenib was approved by the FDA on August 6, 2024, offering a first-in-class option for patients with IDH-mutant gliomas.
FDA Accepts Cabozantinib Application in Advanced Neuroendocrine Tumors
The FDA accepted the filing of a supplemental new drug application of cabozantinib (Cabometyx) for the treatment of neuroendocrine tumors on August 6, 2024.
FDA Clears SeCore CDx as Companion Diagnostic for Afami-cel in Synovial Sarcoma
Also on August 6, 2024, the SeCore™ CDx HLA A Sequencing System gained 510(k) clearance from the FDA as a companion diagnostic for afami-cel when used for the treatment of adult patients with unresectable or metastatic synovial sarcoma.
Phase 1/2 Trial of Silmitasertib in Sarcoma and Brain Tumors to Proceed
On August 6, 2024, the FDA also issued a “study may proceed” letter to Senhwa Biosciences regarding the phase 1/2 study (NCT06541262) of silmitasertib (CX-4945) for the treatment of pediatric and adult patients with relapsed/refractory solid tumors.
FDA Approves Improved Denileukin Diftitox in Cutaneous T-Cell Lymphoma
The FDA approved denileukin diftitox (Lymphir; E7777) for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma who have received at least 1 prior systemic therapy on August 8, 2024.
FDA Greenlights Phase 2 Trial for BRG01 in EBV+ Nasopharyngeal Carcinoma
On August 12, 2024, the FDA approved the start of a phase 2 trial evaluating BRG01, an Epstein-Barr virus (EBV)-specific chimeric antigen receptor T-cell therapy in relapsed/metastatic EBV-positive nasopharyngeal carcinoma.
FDA Clears IND Application of Novel Paclitaxel Administration in Pancreatic Cancer
The FDA granted clearance to the investigational new drug application of PTM-101 in pancreatic ductal adenocarcinoma on August 13, 2024.
Microscopic photorealistic image of tumor cells - Generated with Adobe Firefly
Durvalumab Wins FDA Priority Review in Small Cell Lung Cancer
On August 15, 2024, the FDA granted priority review to the supplemental biologics license application for durvalumab for the treatment of limited-stage SCLC.
FDA Grants Fast Track Designation to Deltacel and Radiation in NSCLC
A fast track designation was granted to Deltacel (KB-GDT-01) in combination with low-dose radiation therapy by the FDA on August 14, 2024, for patients with metastatic NSCLC who have progressed on 2 or more prior lines of standard-of-care therapy.
FDA Considers UGN-102 in Low-Grade NMIBC
Also on August 14, 2024, the FDA received a new drug application seeking the approval of mitomycin for intravesical solution (UGN-102) for treating patients with low-grade, intermediate-risk, non–muscle-invasive bladder cancer.
Neoadjuvant/Adjuvant Durvalumab Scores FDA Approval in Resectable NSCLC
The FDA approved durvalumab with platinum-containing chemotherapy in patients with resectable NSCLC without EGFR mutations or ALK rearrangements on August 15, 2024.
FDA Supports Phase 3 Plan for Amezalpat in Hepatocellular Carcinoma
On August 15, 2024, positive feedback on the combination of amezalpat (TPST-1120), atezolizumab (Tecentriq), and bevacizumab (Avastin) for the first-line treatment of patients with unresectable or metastatic hepatocellular carcinoma were announced.
FDA Grants Priority Review to Vimseltinib Application in Tenosynovial Giant Cell Tumor
The FDA granted priority review to the new drug application of vimseltinib (DCC-3014) for the treatment of tenosynovial giant cell tumor on August 16, 2024.
PT217 Gains FDA Orphan Drug Designation in Neuroendocrine Carcinoma
Also on August 16, 2024, the FDA granted PT217 an orphan drug designation for the treatment of neuroendocrine carcinoma.
FDA Grants T-DXd Breakthrough Therapy Designation in HR+/HER2-Low Breast Cancer
The FDA granted breakthrough therapy designation to T-DXd on August 19, 2024, in unresectable or metastatic HR-positive /HER2-low or HER2-ultralow breast cancer.
Microscopic, photorealistic image of liver tumor cells - Generated with Adobe Firefly
FDA Lifts Partial Clinical Hold on Phase 1 YL202 Trial in NSCLC and Breast Cancer
On August 19, 2024, the FDA also lifted the partial clinical hold placed on the phase 1 YL202-INT-101-01 trial (NCT05653752) of YL202 (BNT326) for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR mutations or HR-positive/HER2-negative breast cancer.
FDA Clears Lazertinib/Amivantamab for First-Line EGFR-Mutated NSCLC
The FDA also approved the combination of lazertinib and amivantamab for the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations on August 19, 2024.
GSK5764227 Gains FDA Breakthrough Therapy Designation in ES-SCLC
The FDA granted breakthrough therapy designation to GSK5764227 (HS-20093) in extensive-stage SCLC with disease progression on or after platinum-based chemotherapy on August 20, 2024.
FDA Issues Complete Response Letter for Linvoseltamab BLA in RRMM
Also on August 20, 2024, a complete response letter has been issued by the FDA to the biologics license application seeking the approval of linvoseltamab (REGN5458) in relapsed/refractory multiple myeloma that has progressed following 3 or more prior therapies.
FDA Accepts Nivolumab/Ipilimumab Application for First-Line HCC Treatment
On August 21, 2024, the FDA accepted the supplemental biologics license application for nivolumab (Opdivo) plus ipilimumab (Yervoy) in the first line for patients with unresectable hepatocellular carcinoma.
FDA Fast Tracks 64Cu-SAR-bisPSMA for Prostate Cancer Imaging
64Cu-SAR-bisPSMA for PET imaging of patients with prostate-specific membrane antigen-positive prostate cancer lesions with suspected metastasis who are candidates for initial definitive therapy received fast track designation from the FDA on August 22, 2024.
APG-157 Earns FDA Fast Track Designation for Head and Neck Cancer Treatment
The FDA granted fast track designation to APG-157, a next-generation immuno-oncology drug, on August 22, 2024, for the neoadjuvant treatment of head and neck cancer.
FDA’S ODAC to Discuss PD-L1 Levels for ICIs in Gastric and Esophageal Cancers
On August 23, 2023, it was announced that the FDA will hold a meeting of the Oncologic Drug Advisory Committee on September 26, 2024, to discuss risks and benefits of PD-L1 expression level cutoffs for immune checkpoint inhibitors in gastric, gastroesophageal junction, and esophageal cancers.
INV724 Earns FDA Orphan Drug and Rare Pediatric Drug Designations in Neuroblastoma
The FDA granted rare pediatric disease designation and orphan drug designation to INV724 for the treatment of neuroblastoma, also on August 23, 2024.
Novel BTK Degrader Earns FDA Fast Track Designation in CLL/SLL
The FDA granted fast track designation to BGB-16673 on August 26, 2024, for patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.
FDA Grants Orphan Drug Designation to Opaganib in Neuroblastoma
On August 26, 2024, the FDA also granted an orphan drug designation to opaganib (Yeliva, ABC294640) for the treatment of patients with neuroblastoma.
TruSight Oncology Test Receives FDA Approval for NTRK and RET Fusion Cancers
The TruSight Oncology Comprehensive test received approval from the FDA as a companion diagnostic to identify patients with solid tumors harboring NTRK gene fusions and NSCLC harboring RET gene fusions on August 27, 2024.
FDA Grants Zongertinib Breakthrough Therapy Designation in HER2-Mutant NSCLC
Also on August 27, 2024, zongertinib (BI 1810631), an investigational oral therapy, was granted breakthrough therapy designation from the FDA in adult patients with advanced, unresectable or metastatic NSCLC harboring HER2 mutations and those who have received a prior systemic therapy.
Mirdametinib Scores FDA Priority Review in Neurofibromatosis Type 1 With PN
On August 28, 2024, the FDA granted priority review to the new drug application of mirdametinib (formerly PD-0325901) in adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.
FDA to Review NDA for TLX101-CDx: A Promising Glioma Imaging Agent
A new drug application was submitted to the FDA on August 28, 2024, for TLX101-CDx (Pixclara; 18F-floretyrosine), an investigational PET agent that helps characterize progressive or recurrent glioma from treatment-related changes.
NXP800 Gains FDA Orphan Drug Status in ARID1a-Deficient Ovarian Cancers
On August 29, 2024, the FDA granted NXP800 orphan drug designation for the potential treatment of patients with ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers.
Navigating ESR1 Mutations in HR-Positive Breast Cancer With Dr Wander
October 31st 2024In this episode of Targeted Talks, Seth Wander, MD, PhD, discusses the clinical importance of ESR1 mutations in HR-positive metastatic breast cancer and how these mutations influence treatment approaches.
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