FDA Makes Strides in Oncology: New Approvals and Promising Developments in March 2024

The FDA made significant progress on various oncology fronts in March 2024 with the approvals of new agents, investigational new drug application approvals for drugs targeting various cancers, and orphan drug designations for several potential therapies.

Notably, amivantamab-vmjw (Rybrevant) with chemotherapy was approved by the FDA for EGFR exon 20 insertion mutation-positive non-small cell lung cancer (NSCLC).

The Oncologic Drug Advisory Committee (ODAC) met 3 times this month where first, they voted that the benefits of imetelstat outweigh the risks for those with lower-risk myelodysplastic syndromes with anemia who are transfusion-dependent and ineligible for erythropoiesis stimulating agents. Then, they discussed idecabtagene vicleucel (ide-cel; Abecma) and decided that the risk/benefit assessment for the proposed indication is favorable for the treatment of adult patients with relapsed/refractory multiple myeloma who have received an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody. The ODAC also found that ciltacabtagene autoleucel (cilta-cel; Carvykti) had a favorable risk/benefit balance for patients with mutliple myeloma.

Additionally, the FDA approvedtislelizumab-jsgr (Tevimbra) for esophageal squamous cell carcinoma and mirvetuximab soravtansine-gynx (Elahere) for platinum-resistant ovarian cancer.

Here is a look back at all the FDA happenings of March 2024.

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FDA Greenlights IND of JAB-30300 for Advanced Solid Tumors

On March 1, 2024, JAB-30300, a P53 Y220C activator, received approval from the FDA for its investigational new drug application. A phase 1/2a clinical trial to investigate its safety and efficacy against advanced solid tumors will begin.

FDA Clears IND of CLN-619 in R/R Multiple Myeloma

The FDA cleared the investigational new drug application for CLN-619, a potential first-in-class humanized IgG1 monoclonal antibody, for patients with relapsed/refractory multiple myeloma on March 1, 2024.

FDA Approves Amivantamab/Chemo in Advanced EGFR Exon20Ins Mut+ NSCLC

Also on March 1, 2024, the FDA approved amivantamab (Rybrevant) with carboplatin and pemetrexed as a first-line treatment in patients with EGFR exon 20 insertion mutation-positive NSCLC.

FDA Lifts Clinical Hold on TIL Therapy Trial in Lung Cancer

The FDA announced it will lift the partial clinical hold on the IOV-LUN-202 trial (NCT04614103) investigating the tumor infiltrating lymphocyte cell therapy LN-145 for the treatment of NSCLC on March 4, 2024.

FDA Grants Orphan Drug Designation to Novel CAR T-Cell Therapy in Colorectal Cancer

The FDA granted A2B530, a novel cell therapy, orphan drug designation on March 4, 2024, for the treatment of patients with colorectal cancer with germline heterozygous HLA-A*02-positive disease expressing carcinoembryonic antigen and lost HLA-A*02 expression.

FDA Initiates Rolling NDA of Mirdametinib in NF1-PN

On March 4, 2024, the FDA initiated a rolling submission of a new drug application for the mirdametinib (formerly PD-0325901) for the treatment of pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas.

FDA Clears Clinical Study of Vaccine Therapy for Ovarian Cancer

The FDA granted clearance to a first-in-human phase 1 clinical trial of Innocell, an investigational autologous vaccine therapy, for the treatment of stage III/IV ovarian cancer on March 5, 2024.

Olaptesed Pegol Gains FDA Clearance in Glioblastoma

On March 5, 2024, the FDA cleared the investigational new drug application for olaptesed pegol (NOX-A12) as a potential treatment for adult patients with aggressive brain cancer.

FDA Grants Orphan Drug Designation to Avutometinib in Ovarian Cancer

The FDA also granted an orphan drug designation to avutometinib with or without defactinib on March 5, 2024, for the treatment of patients with recurrent low-grade serous ovarian cancer.

FDA Approves Inotuzumab for Pediatric Patients with ALL

On March 6, 2024, the FDA approved inotuzumab ozogamicin (Besponsa) for the treatment of patients 1 year or older with relapsed/refractory CD22-positive B-cell precursor acute lymphoblastic leukemia.

Cambritaxestat Wins FDA Orphan Drug Designation in Pancreatic Cancer

An orphan drug designation was granted to the autotaxin inhibitor cambritaxestat by the FDA for the treatment of patients with pancreatic cancer on March 7, 2024.

Nivolumab With Chemotherapy Gains FDA Approval in Urothelial Cancer

On March 7, 2024, the FDA also approved nivolumab (Opdivo) plus cisplatin-based chemotherapy for the first-line treatment of patients with previously untreated unresectable or metastatic urothelial carcinoma.

FDA Lifts Clinical Hold on Trial of NX-2127 in B-Cell Malignancies

The FDA lifted the partial clinical hold on the phase 1 trial of NX-2127 (NCT04830137) for the treatment of patients with relapsed or refractory B-cell malignancies on March 11, 2024.

TERN-701 Gains FDA Orphan Drug Designation in Chronic Myeloid Leukemia

The FDA granted TERN-701 an orphan drug designation on March 11, 2024, for the treatment of chronic myeloid leukemia.

FDA Accepts Ensartinib Application in Non-Small Cell Lung Cancer

The new drug application of ensartinib for the first-line treatment of patients with anaplastic lymphoma kinase-positive NSCLC was accepted by the FDA on March 13, 2024.

LYT-200 Gains FDA Orphan Drug Designation in Acute Myeloid Leukemia

On March 13, 2024, an orphan drug designation from the FDA was granted to LYT-200 for the treatment of patients with acute myeloid leukemia.

P-BCMA-ALLO1 Gains FDA Orphan Drug Designation in R/R Multiple Myeloma

P-BCMA-ALLO1 for relapsed/refractory multiple myeloma received orphan drug designation from the FDA on March 13, 2024.

FDA ODAC Votes Yes on Imetelstat for Patients with MDS

On March 14, 2024, the FDA’s ODAC voted that the benefits of imetelstat outweigh the risks for patients with lower-risk myelodysplastic syndromes with anemia who are transfusion-dependent and ineligible for erythropoiesis stimulating agents.

FDA Approves Tislelizumab for Unresectable or Metastatic ESCC

The FDA approved tislelizumab for treating esophageal squamous cell carcinoma after prior chemotherapy in patients who haven't received a PD-1/PD-L1 inhibitor on March 14, 2024.

FDA’s ODAC Finds Cilta-Cel Favorable in R/R Multiple Myeloma

The FDA’s ODAC voted on March 15, 2024, that the benefits of cilta-cel do outweigh the risks for patients with multiple myeloma who have already undergone multiple prior treatment lines.

FDA’s ODAC Votes Benefits of Ide-Cel Outweigh Risks in R/R Multiple Myeloma

Also on March 15, 2024, the FDA’s ODAC voted that the risk/benefit assessment for ide-cel for the proposed indication is favorable.

FDA Accepts BLA Resubmission of Denileukin Diftitox for R/R CTCL

The FDA accepted Citius Pharmaceuticals’ resubmitted biologics license application on March 18, 2024, for denileukin diftitox as a treatment for relapsed/refractory cutaneous T-cell lymphoma.

FDA Grants Accelerated Approval to Ponatinib/Chemotherapy for Ph+ ALL

The FDA granted accelerated approval to the combination of ponatinib (Iclusig) with chemotherapy for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.

FDA Grants Fast Track Designation to MVR-T3011 in HNSCC

MVR-T3011 received a fast track designation from the FDA for the treatment of recurrent or metastatic head and neck squamous cell cancer.

FDA Fast Tracks PT886 in CLDN18.2+ Pancreatic Cancer

The FDA has granted a fast track designation to PT886, a first-in-class native IgG-like bispecific antibody, for metastatic claudin 18.2-positive pancreatic adenocarcinoma treatment on March 20, 2024.

FDA Approves Safety Label Changes for Fluorouracil Injection

On March 21, 2024, the FDA updated the label of fluorouracil to warn about serious and potentially fatal adverse effects in patients with a genetic condition called dihydropyrimidine dehydrogenase deficiency.

FDA Grants Full Approval to Mirvetuximab for Platinum-Resistant Ovarian Cancer

Mirvetuximab soravtansine (Elahere) was fully approved by the FDA for patients with platinum-resistant ovarian cancer with high folate receptor alpha expression who have received 1 to 3 prior lines of treatment on March 22, 2024.

FDA Clears IND of BAT8006 in Platinum-Resistant Ovarian Cancers

The FDA cleared the investigational new drug application for the antibody-drug conjugate BAT8006 on March 25, 2024, for platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA Denies Approval of Odronextamab in DLBCL and Follicular Lymphoma

On March 25, 2024, the FDA issued 2 complete response letters for the biologics license application of odronextamab (REGN1979) for patients with relapsed/refractory follicular lymphoma and relapsed/refractory diffuse large B-cell lymphoma.

Revumenib Earns FDA Priority Review in R/R KMT2Ar Acute Leukemia

Priority review from the FDA was granted to the new drug application for revumenib in adult and pediatric relapsed or refractory KMT2A-rearranged acute leukemia on March 26, 2024.

FDA Clears IND for NST-628 for Advanced Solid Tumors With RAS-MAPK Mutations

The FDA cleared the investigational new drug application for NST-628 for the treatment of patients with advanced solid tumors with genetic alterations in the RAS-MAPK pathway on March 28, 2024.