News|Articles|September 2, 2025
A Month of Breakthroughs: The FDA's Oncology Decisions in August 2025
Author(s)Targeted Oncology Staff
Fact checked by: Sabrina Serani
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Key Takeaways
- The FDA approved several targeted therapies, emphasizing precision medicine and addressing specific mutations in challenging cancers.
- Notable advancements included treatments for rare brain tumors and new avenues for lung and prostate cancers.
August 2025 marked a transformative month in oncology as the FDA accelerates approvals for innovative cancer therapies, enhancing treatment options for patients.
August 2025 proved to be a pivotal month for oncology, with the US FDA advancing treatment options for several challenging cancers. The month's announcements underscored the ongoing shift toward precision medicine, with accelerated approvals for targeted therapies addressing specific mutations in difficult-to-treat diseases. From breakthroughs in rare brain tumors to new treatment avenues for common lung and prostate cancers, the FDA's decisions have opened new doors for patients and clinicians. Here are the key approvals and noteworthy developments that shaped the oncology landscape throughout the month.
FDA Greenlights Novel Colorectal Cancer Triplet Therapy Trial
On August 4, Anbogen Therapeutics received FDA approval for its investigational new drug (IND) application for ABT-301, paving the way for a phase 1/2 clinical trial in patients with metastatic colorectal cancer (mCRC).
FDA Grants Priority Review for Liso-Cel in Marginal Zone Lymphoma
On August 5, the FDA accepted for priority review the supplemental biologics license application (sBLA) for lisocabtagene maraleucel (liso-cel; Breyanzi), a CD19-directed chimeric antigen receptor (CAR) T-cell therapy, in marginal zone lymphoma (MZL). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 5, 2025.
FDA Clears IND for GLIX1 in Glioblastoma, Phase 1 Study to Proceed
Also on August 5, the FDA cleared the investigational new drug (IND) application of GLIX1, a first-in-class small molecule targeting DNA repair vulnerabilities, offering a potential therapy for glioblastoma.
Dordaviprone Receives Accelerated FDA Approval for Diffuse Midline Glioma
On August 6, the FDA granted accelerated approval to dordaviprone (Modeyso), a novel systemic therapy for the treatment of diffuse midline glioma (DMG) with an H3 K27M mutation.
FDA Clears Novel Combination Trial for Recurrent Glioblastoma
Also on August 6, the FDA approved an IND for phase Ib/2a clinical trial evaluating the combination of the novel agent STAR-001 (LP-184) with the FDA-approved drug spironolactone for the treatment of glioblastoma (GBM). The trial will focus on adult patients whose GBM has progressed after initial treatment.
FDA Expands Approval for Tocilizumab Biosimilar to Treat CRS
On August 6, the FDA approved an expanded indication for the intravenous (IV) formulation of tocilizumab-anoh (Avtozma), a biosimilar to tocilizumab (Actemra), to include the treatment of cytokine release syndrome (CRS) in adults and pediatric patients aged 2 years and older.
FDA Fast Track Designation Granted for Novel BTK/LYN Dual Inhibitor in CLL/SLL
On August 6, the FDA granted fast track designation to birelentinib (DZD8586), an investigational, first-in-class, noncovalent dual inhibitor of LYN and BTK. The designation is for the treatment of adult patients with relapsed/refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.
FDA Accepts NDA of New F 18 Formulation for Prostate Cancer Imaging
On August 6, the FDA accepted a new drug application (NDA) for a new formulation of piflufolastat F 18 (Pylarify), an advanced imaging agent used for PET scans in patients with prostate cancer.
FDA Approves PEG Hydrogel Embolic System for Hypervascular Tumors
On August 7, the Embrace Hydrogel Embolic System (HES), a polyethylene glycol (PEG) hydrogel embolic system, received US premarket approval for the embolization of hypervascular tumors in peripheral arteries, marking a significant advancement in interventional oncology.
Roxadustat Phase 3 Trial for Anemia in Lower-Risk MDS Receives FDA Go-Ahead
The development of roxadustat (Evrenzo) has taken a significant step forward for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) who have a high red blood cell (RBC) transfusion burden. Following a positive Type C meeting with the FDA, FibroGen, sponsor, plans to submit a phase 3 trial protocol in the fourth quarter of 2025.
PQ203 Receives Regulatory Clearance for Breast Cancer Trial
On August 7, regulatory agencies in the United States and Canada granted clearance for a phase 1 clinical trial of PQ203, a novel peptide drug conjugate, in patients with advanced triple-negative breast cancer (TNBC), and the US FDA granted the agent fast track designation.
FDA Grants Accelerated Approval to Zongertinib in HER2 NSCLC
On August 8, the FDA granted accelerated approval to zongertinib (Hernexeos) for the treatment of patients with unresectable or metastatic nonsquamous non–small cell lung cancer (NSCLC) with tumors harboring a HER2 tyrosine kinase domain (TKD) activating mutation and who have received prior systemic therapy.
FDA Accepts NDA for Vepdegestrant in ER+/HER2- Metastatic Breast Cancer
On August 11, the FDA has accepted an NDA for vepdegestrant, an investigational oral PROteolysis TArgeting Chimera (PROTAC) being developed by Arvinas and Pfizer for the treatment of ER+, HER2–, ESR1-mutated advanced or metastatic breast cancer who have progressed on prior endocrine-based therapy.
HLD-0915 Receives FDA Fast Track Designation for mCRPC Treatment
On August 14, the FDA granted fast track designation to HLD-0915, a novel bifunctional small molecule therapy, for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC). This designation aims to accelerate the development and review of the investigational drug, which is currently being evaluated in a phase 1/2 clinical trial (NCT06800313).
Izalontamab Brengitecan Earns FDA Breakthrough Designation in EGFR+ NSCLC
On August 18, the FDA granted breakthrough therapy designation to izalontamab brengitecan (iza-bren), a bispecific antibody-drug conjugate (ADC), for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR mutations.
Ifinatamab Deruxtecan Receives FDA Breakthrough Therapy Designation in SCLC
On August 18, the FDA granted breakthrough therapy designation to ifinatamab deruxtecan (I-DXd), a B7-H3–directed DXd ADC for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy.
FDA Clears Phase 1/2 Study for Novel CDH17-Targeting ADC in Advanced GI Cancers
On August 19, the FDA granted IND clearance for 7MW4911, a first-in-class CDH17-targeting ADC. This regulatory milestone enables the company to initiate a phase 1/2 clinical trial to evaluate the safety, pharmacokinetics, and preliminary efficacy of the drug in patients with advanced colorectal cancer and other advanced gastrointestinal (GI) tumors.
FDA Approves New Companion Dx, Advancing Precision in Colorectal Cancer
On August 20, in a step for personalized cancer care, the FDA approved Agilent Technologies Inc's MMR IHC Panel pharmDx (Dako Omnis), a new companion diagnostic (CDx) test for CRC. This approval is poised to provide oncologists and pathologists with an essential tool for identifying patients who will most likely benefit from specific immunotherapy treatments.
Phase 3 Trial Initiated for Novel Bispecific Antibody in NSCLC
On August 24, the FDA has cleared an IND application to begin a global phase 3 clinical trial of IBI363, a novel bispecific antibody, in patients with immunotherapy (IO)-resistant squamous NSCLC. This marks the first global phase 3 study for the agent, which is a PD-1/IL-2α-biased bispecific antibody fusion protein.
FDA Grants Breakthrough Designation for ctDNA Test in Colorectal Cancer
On August 25, the FDA granted breakthrough device designation to the Haystack MRD test, a circulating tumor DNA (ctDNA) assay developed by Quest Diagnostics. This designation recognizes the test's potential to significantly improve the management of minimal residual disease (MRD) in patients with stage II CRC following curative-intent surgical treatment.
FDA Breakthrough Therapy Granted for Novel ADC in Endometrial Cancer
On August 26, the FDA granted breakthrough therapy designation to rinatabart sesutecan (Rina-S; GEN1184), an investigational folate receptor alpha (FRα)-directed ADC, for the treatment of adult patients with recurrent or progressive endometrial cancer.This designation applies to patients whose disease has progressed on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy.
FDA Accepts Gedatolisib for Real-Time Review in HR+/HER2– Advanced Breast Cancer
On August 27, the FDA agreed to review an NDA for gedatolisib, an investigational multitarget pan-PI3K and mTORC1/2 inhibitor, under its Real-Time Oncology Review (RTOR) program.
TT125-802 Gains FDA Fast Track Designation in NSCLC
On August 28, a novel small-molecule bromodomain inhibitor, TT125-802, received 2 fast track designations from the FDA for the treatment of NSCLC.
FDA Grants Orphan Status to Novel JAK2 Inhibitor for Polycythemia Vera
On August 28, the FDA granted orphan drug designation to VGT-1849B, an investigational selective JAK2 inhibitor, for the treatment of polycythemia vera (PV).
FDA Approves 3-Month Leuprolide Mesylate Formulation for Advanced Prostate Cancer
On August 29, the US FDA granted approval for a 3-month, ready-to-use, long-acting injectable (LAI) formulation of leuprolide mesylate (Camcevi ETM) for the treatment of adult patients with advanced prostate cancer.
August 2025 proved to be a pivotal month for oncology, with the US FDA advancing treatment options for several challenging cancers. The month's announcements underscored the ongoing shift toward precision medicine, with accelerated approvals for targeted therapies addressing specific mutations in difficult-to-treat diseases. From breakthroughs in rare brain tumors to new treatment avenues for common lung and prostate cancers, the FDA's decisions have opened new doors for patients and clinicians. Here are the key approvals and noteworthy developments that shaped the oncology landscape throughout the month.
On August 4, Anbogen Therapeutics received FDA approval for its investigational new drug (IND) application for ABT-301, paving the way for a phase 1/2 clinical trial in patients with metastatic colorectal cancer (mCRC).
On August 5, the FDA accepted for priority review the supplemental biologics license application (sBLA) for lisocabtagene maraleucel (liso-cel; Breyanzi), a CD19-directed chimeric antigen receptor (CAR) T-cell therapy, in marginal zone lymphoma (MZL). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 5, 2025.
Also on August 5, the FDA cleared the investigational new drug (IND) application of GLIX1, a first-in-class small molecule targeting DNA repair vulnerabilities, offering a potential therapy for glioblastoma.
On August 6, the FDA granted accelerated approval to dordaviprone (Modeyso), a novel systemic therapy for the treatment of diffuse midline glioma (DMG) with an H3 K27M mutation.
Also on August 6, the FDA approved an IND for phase Ib/2a clinical trial evaluating the combination of the novel agent STAR-001 (LP-184) with the FDA-approved drug spironolactone for the treatment of glioblastoma (GBM). The trial will focus on adult patients whose GBM has progressed after initial treatment.
On August 6, the FDA approved an expanded indication for the intravenous (IV) formulation of tocilizumab-anoh (Avtozma), a biosimilar to tocilizumab (Actemra), to include the treatment of cytokine release syndrome (CRS) in adults and pediatric patients aged 2 years and older.
On August 6, the FDA granted fast track designation to birelentinib (DZD8586), an investigational, first-in-class, noncovalent dual inhibitor of LYN and BTK. The designation is for the treatment of adult patients with relapsed/refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.
On August 6, the FDA accepted a new drug application (NDA) for a new formulation of piflufolastat F 18 (Pylarify), an advanced imaging agent used for PET scans in patients with prostate cancer.
On August 7, the Embrace Hydrogel Embolic System (HES), a polyethylene glycol (PEG) hydrogel embolic system, received US premarket approval for the embolization of hypervascular tumors in peripheral arteries, marking a significant advancement in interventional oncology.
The development of roxadustat (Evrenzo) has taken a significant step forward for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) who have a high red blood cell (RBC) transfusion burden. Following a positive Type C meeting with the FDA, FibroGen, sponsor, plans to submit a phase 3 trial protocol in the fourth quarter of 2025.
On August 7, regulatory agencies in the United States and Canada granted clearance for a phase 1 clinical trial of PQ203, a novel peptide drug conjugate, in patients with advanced triple-negative breast cancer (TNBC), and the US FDA granted the agent fast track designation.
On August 8, the FDA granted accelerated approval to zongertinib (Hernexeos) for the treatment of patients with unresectable or metastatic nonsquamous non–small cell lung cancer (NSCLC) with tumors harboring a HER2 tyrosine kinase domain (TKD) activating mutation and who have received prior systemic therapy.
On August 11, the FDA has accepted an NDA for vepdegestrant, an investigational oral PROteolysis TArgeting Chimera (PROTAC) being developed by Arvinas and Pfizer for the treatment of ER+, HER2–, ESR1-mutated advanced or metastatic breast cancer who have progressed on prior endocrine-based therapy.
On August 14, the FDA granted fast track designation to HLD-0915, a novel bifunctional small molecule therapy, for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC). This designation aims to accelerate the development and review of the investigational drug, which is currently being evaluated in a phase 1/2 clinical trial (NCT06800313).
On August 18, the FDA granted breakthrough therapy designation to izalontamab brengitecan (iza-bren), a bispecific antibody-drug conjugate (ADC), for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR mutations.
On August 18, the FDA granted breakthrough therapy designation to ifinatamab deruxtecan (I-DXd), a B7-H3–directed DXd ADC for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy.
On August 19, the FDA granted IND clearance for 7MW4911, a first-in-class CDH17-targeting ADC. This regulatory milestone enables the company to initiate a phase 1/2 clinical trial to evaluate the safety, pharmacokinetics, and preliminary efficacy of the drug in patients with advanced colorectal cancer and other advanced gastrointestinal (GI) tumors.
On August 20, in a step for personalized cancer care, the FDA approved Agilent Technologies Inc's MMR IHC Panel pharmDx (Dako Omnis), a new companion diagnostic (CDx) test for CRC. This approval is poised to provide oncologists and pathologists with an essential tool for identifying patients who will most likely benefit from specific immunotherapy treatments.
On August 24, the FDA has cleared an IND application to begin a global phase 3 clinical trial of IBI363, a novel bispecific antibody, in patients with immunotherapy (IO)-resistant squamous NSCLC. This marks the first global phase 3 study for the agent, which is a PD-1/IL-2α-biased bispecific antibody fusion protein.
On August 25, the FDA granted breakthrough device designation to the Haystack MRD test, a circulating tumor DNA (ctDNA) assay developed by Quest Diagnostics. This designation recognizes the test's potential to significantly improve the management of minimal residual disease (MRD) in patients with stage II CRC following curative-intent surgical treatment.
On August 26, the FDA granted breakthrough therapy designation to rinatabart sesutecan (Rina-S; GEN1184), an investigational folate receptor alpha (FRα)-directed ADC, for the treatment of adult patients with recurrent or progressive endometrial cancer.This designation applies to patients whose disease has progressed on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy.
On August 27, the FDA agreed to review an NDA for gedatolisib, an investigational multitarget pan-PI3K and mTORC1/2 inhibitor, under its Real-Time Oncology Review (RTOR) program.
On August 28, a novel small-molecule bromodomain inhibitor, TT125-802, received 2 fast track designations from the FDA for the treatment of NSCLC.
On August 28, the FDA granted orphan drug designation to VGT-1849B, an investigational selective JAK2 inhibitor, for the treatment of polycythemia vera (PV).
On August 29, the US FDA granted approval for a 3-month, ready-to-use, long-acting injectable (LAI) formulation of leuprolide mesylate (Camcevi ETM) for the treatment of adult patients with advanced prostate cancer.
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