September 2024 FDA Roundup: Key Decisions Drive Oncology Forward

Microscopic, photorealistic image of tumor cells - Generated with Adobe Firefly

In September 2024, the FDA made several significant strides in oncology. Notably, the agency approved a subcutaneous formulation of atezolizumab (Tecentriq) for various cancer types, offering a more convenient administration option for patients. Additionally, ribociclib (Kisqali) was granted approval for early-stage breast cancer, expanding treatment options for this disease. Other notable approvals included isatuximab (Sarclisa) for multiple myeloma, selpercatinib (Retevmo) for medullary thyroid cancer, and osimertinib (Tagrisso) for advanced non–small cell lung cancer (NSCLC). These advancements demonstrate the FDA's ongoing commitment to improving cancer treatments and enhancing patient outcomes.

FDA Grants RMAT Designation to Novel TIL Therapy OBX-115 in Melanoma

On September 3, OBX-115, a novel TIL therapy, was granted FDA regenerative medicine advanced therapy (RMAT) designation in unresectable or metastatic melanoma resistant to immune checkpoint inhibitor therapy. In July 2024, the FDA also granted OBX-115 fast track designation for the same indication.

FDA Oks FoundationOne CDx and Liquid CDx for Use With Olaparib/Abiraterone in Prostate Cancer

The FDA approved the FoundationOne CDx and FoundationOne Liquid CDx as companion diagnostics for the combination of olaparib (Lynparza) and abiraterone (Zytiga) with prednisone or prednisolone for the treatment of adult patients with BRCA-mutated metastatic castration-resistant prostate cancer, also on September 3.

FDA Approves Ready-to-Use Subcutaneous Bortezomib Formulation

On September 6, the first ready-to-use administration of subcutaneous bortezomib (Boruzu) was approved by the FDA. The new formulation minimizes the compounding preparation required for standard bortezomib administration and is anticipated to launch in the second quarter of 2025.

Novel Bispecific Antibody Fusion Protein Earns FDA Fast Track in Melanoma

The FDA granted fast track designation to IBI363, an investigational PD-1/IL-2α bispecific antibody fusion protein, for the treatment of unresectable locally advanced or metastatic melanoma that has progressed following at least 1 line of systemic therapy, including a PD-(L)1 inhibitor, also on September 6.

VMT01 Earns FDA Fast Track Designation in MC1R-Positive Melanoma

On September 5, the FDA granted fast track designation to 212Pb VMT01 for the diagnosis and treatment of unresectable or metastatic melanoma with MC1R tumor expression. The agent is designed to target and deliver 212Pb to tumor sites expressing MC1R.

Microscopic, photorealistic image of tumor cells - Generated with Adobe Firefly

FDA Grants Orphan Drug Designation to ABD-147 in Neuroendocrine Carcinoma

Orphan drug designation was granted to ABD-147 for the treatment of neuroendocrine carcinoma, also on September 5. A first-in-human study will evaluate ABD-147 in patients with small cell lung cancer (SCLC) or large cell neuroendocrine carcinoma who previously received platinum-based therapy.

Certepetide Wins FDA Orphan Drug Designation in Cholangiocarcinoma

Also on September 5, orphan drug designation was also given to certepetide (formerly LSTA1) for the treatment of cholangiocarcinoma. Certepetide is an investigational agent that assists coadministered anticancer drugs to more effectively penetrate tumors.

Tebapivat Scores FDA Orphan Drug Designation in MDS-Associated Anemia

Tebapivat (AG-946), a novel pyruvate kinase activator, earned orphan drug designation for the treatment of myelodysplastic syndromes-associated anemia on September 11. A phase 2b study (NCT05490446) is currently evaluating the agent.

FDA Grants Elraglusib Orphan Drug Designation in Soft Tissue Sarcoma

Also on September 11, the FDA granted orphan drug designation to elraglusib, a novel GSK-3β inhibitor, for the treatment of patients with soft tissue sarcoma.

FDA Clears IND of VNX-101 in CD19+ Acute Lymphoblastic Leukemia

The FDA cleared an investigational new drug application for the gene therapy product VNX-101 for the treatment of CD19-positive acute lymphoblastic leukemia on September 12.

FDA Approves New Subcutaneous Atezolizumab Formulation

A subcutaneous formulation of atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) was approved for all adult indications of the intravenous version of the agent on September 12. These include NSCLC, SCLC, hepatocellular carcinoma (HCC), melanoma, and alveolar soft part sarcoma.

FDA Grants P-BCMA-ALLO1 RMAT Designation in R/R Multiple Myeloma

On September 16, RMAT designation was given to P-BCMA-ALLO1 for the potential treatment of relapsed/refractory (R/R) multiple myeloma.P-BCMA-ALLO1 is an investigational stem cell memory T-cell (TSCM)-based allogeneic chimeric antigen receptor (CAR) T-cell therapy.

FDA Approves Ribociclib With an Aromatase Inhibitor in Early Breast Cancer

The FDA approved adjuvant ribociclib plus an aromatase inhibitor in hormone receptor-positive, HER2-negative stage II and III early breast cancer at high risk of recurrence on September 17. Data from the phase 3 NATALEE trial (NCT03701334) support this approval.

FDA Approves Frontline Pembrolizumab/Chemotherapy in Advanced Mesothelioma

Also on September 17, the FDA approved the frontline combination of pembrolizumab (Keytruda) and chemotherapy in patients with unresectable advanced or metastatic malignant pleural mesothelioma. This approval is supported by findings from the 2/3 KEYNOTE-483 trial (NCT02784171).

Microscopic, photorealistic image of tumor cells - Generated with Adobe Firefly

FDA Grants Fast Track Designation to ICT01 Plus Azacitidine and Venetoclax in AML

ICT01, a humanized anti-BTN3A monoclonal antibody, plus azacitidine and venetoclax (Venclexta) earned fast track designation for the treatment of acute myeloid leukemia in patients 75 or older or those who have comorbidities that preclude standard induction chemotherapy on September 18.

FDA Grants Orphan Drug Designation to CF33-hNIS in Cholangiocarcinoma

On September 18, orphan drug designation was granted to CF33-hNIS (Vaxinia) for the treatment of patients with cholangiocarcinoma. CF33-hNIS is a novel oncolytic virotherapy under investigation in the MAST trial (NCT05346484).

FDA Approves New Dosage of Trastuzumab Biosimilar for HER2-Overexpressing Cancers

Also on September 18, the FDA approved the 420-mg strength of the trastuzumab (Herceptin) biosimilar trastuzumab-strf (Hercessi; formerly HLX02). In April 2024, the FDA approved the 150-mg dose of trastuzumab-strf.

FDA Clears Amivantamab/Chemotherapy Combo in EGFR-Mutant NSCLC

Amivantamab-vmjw (Rybrevant) plus carboplatin and pemetrexed were approved on September 19 for the treatment of locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations. The phase 3 MARIPOSA-2 trial (NCT04988295) supports this approval.

FDA Clears Investigational New Drug Application for CD-001 in Oncology

On September 20, the FDA granted clearance to the investigational new drug application of CD-001. With this, CD-001 will enter a first-in-human, phase 1 trial.

FDA Approves Isatuximab Plus VRd in Transplant-Ineligible Multiple Myeloma

Isatuximab plus bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (VRd) were approved for the treatment of patients with transplant-ineligible, newly diagnosed multiple myeloma, also on September 20. This marked the first approval of anti-CD38 therapy in combination with standard-of-care VRd in this patient population.

FDA Grants FTD to EO-3021 in Advanced Gastric and GEJ Cancers Expressing Claudin 18.2

On September 23, the FDA granted fast track designationto EO-3021 in advanced or metastatic gastric and gastroesophageal junction (GEJ) cancer expressing Claudin18.2 that has progressed on or after prior therapy.

FDA Receives Resubmitted NDA for Camrelizumab/Rivoceranib Combo in Unresectable HCC

Also on September 23, theFDA received a resubmitted new drug application (NDA) seeking the approval of camrelizumab plus rivoceranib for the frontline treatment of patients with unresectable HCC. In May 2024, the FDA issued a complete response letter for the original NDA.

LP-184 Earns 3 FDA Rare Pediatric Disease Designations in Solid Tumors

The FDA granted 3 rare pediatric disease designations (RPDDs) to the investigational agent LP-184 in malignant rhabdoid tumors, rhabdomyosarcoma, and hepatoblastoma, also on September 23. LP-184 previously earned a RPDD in atypical teratoid rhabdoid tumors.

FDA Approves Osimertinib in Stage III EGFR-Mutated NSCLC

On September 25, the FDA approved osimertinib for the treatment of patients with stage III EGFR-mutated NSCLC whose disease did not progress during or following concurrent or sequential platinum-based chemoradiation.The approval was supported by the phase 3 LAURA trial (NCT03521154).

FDA ODAC Finds Limited Benefit for Checkpoint Inhibitors in Low PD-L1 Gastric Cancer

The FDA’s ODAC voted 2 to 10 that the risk-benefit assessment was not favorablefor the use of checkpoint inhibitors in first-line advanced HER2-negative gastric and GEJ adenocarcinoma in patients with PD-L1 expression less than 1 in the morning session of the Committee meeting on September 26.

FDA ODAC Votes Against Checkpoint Inhibitors in PD–L1-Negative Esophageal Cancer

In the afternoon session of the Committee meeting, the ODAC voted 11 to 1 that the risk-benefit assessment was not favorablefor the use of checkpoint inhibitors in patients with metastatic or unresectable esophageal squamous cell carcinoma with PD-L1 expression less than 1.

sNDA Filed With FDA for Darolutamide Plus ADT in mHSPC

A supplemental NDA seeking expanded indication for darolutamide (Nubeqa) given with androgen deprivation therapy in metastatic hormone-sensitive prostate cancer was submitted to the FDA, also on September 26. Data from the phase 3 ARANOTE trial (NCT04736199) support this submission.

FDA Grants Traditional Approval to Selpercatinib in RET+ Medullary Cancer

On September 27, the FDA granted traditional approval to selpercatinib for the treatment of adult and pediatric patients aged 2 years and older with advanced or metastatic medullary thyroid cancer with a RET mutation, as detected by an FDA-approved test, who require systemic therapy.