A Look Back at FDA News From March 2023

In March 2023, the FDA brought 5 approvals to the market for patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma, BRAF V600E-mutated low-grade glioma, metastatic or recurrent locally advanced Merkel cell carcinoma, microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, and to decrease chemotherapy-associated febrile neutropenia in patients with cancer.

The FDA also widened the indication of 3 drugs for patients with hormone receptor-positive, HER2-negative, node-positive, early breast cancer at a high risk of recurrence, non–small cell lung cancer, and metastatic prostate cancer.

During an FDA’s Oncologic Drugs Advisory Committee (ODAC) meeting to discuss polatuzumab vedotin-piiq (Polivy) injection, the committee ultimately voted 11 to 2 that the benefit/risk profile of for the agent was favorable for the treatment of previously untreated diffuse large B-cell lymphoma (DLBCL).

While the FDA placed a clinical hold on the phase 1 trial (NCT05514717) of XMT-2056, they lifted the partial clinical hold that was previously placed on the phase 1/2 VELA trial (NCT05252416) evaluating BLU-222 for patients with solid tumors.

Additionally, multiple drugs were granted orphan drug status in March,1 drug was granted breakthrough therapy designation, and 1 drug was granted an investigational new drug application for advanced solid tumors and lymphomas. Additionally, a couple of new drug applications were submitted to the FDA in March.

Here’s a look back at the notable FDA updates from March 2023.

FDA to Review BLA for Perioperative Pembrolizumab/Chemotherapy for Stage II-IIIB NSCLC

At the start of March, the FDA accepted a biologics license application for pembrolizumab (Keytruda) for the treatment of patients with resectable stage II, IIIA, or IIIB (T3-4N2) non–small cell lung cancer (NSCLC) in combination with platinum containing chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment.

FDA Receives NDA for Niraparib Plus Abiraterone and Prednisone for BRCA+ mCRPC

Also on March 1, a new drug application (NDA) was submitted to the FDA seeking the approval of niraparib (Zejula) in combination with abiraterone acetate, in the form of a dual-action tablet, plus prednisone, for patients with BRCA-positive metastatic castration-resistant prostate cancer.

Novel CEACAM Antibody Lands FDA Orphan Drug Designation for HCC

The FDA also granted an orphan drug designation to YB-200 for the treatment of patients with hepatocellular carcinoma on March 1.

FDA Considers Approval of Cosibelimab for Advanced Cutaneous Squamous Cell Carcinoma

On March 2, the FDA accepted a biologic license application for cosibelimab (CK-301), an anti-PD-L1 antibody and potential treatment option, for patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or radiation.

FDA Gives Orphan Drug Designation to Temferon for Glioblastoma Multiforme

The FDA provided orphan drug status for the cell therapy Temferon for the treatment of patients with glioblastoma multiforme, also on March 2.

FDA Widens Indication for Abemaciclib in HR+/HER2–, Node+, High-Risk Breast Cancer

The FDA expanded the indication for abemaciclib (Verzenio), in combination with endocrine therapy, for the adjuvant treatment of adult patients with HR-positive, HER2-negative, node-positive, early breast cancer at a high risk of recurrence on March 3.

FDA OKs Pegfilgrastim Biosimilar to Decrease Chemo-Associated Febrile Neutropenia

The FDA granted approval to pegfilgrastim-cbqv (Udencya), a pegfilgrastim biosimilar, in order to decrease chemotherapy-associated febrile neutropenia in patients with cancer, on March 6.

FDA Approves VENTANA PD-L1 Assay as a Cemiplimab CDx in NSCLC

On March 6, the FDA also expanded the label for the VENTANA PD-L1 (SP263) Assay. The test is now an approved companion diagnostic to identify patients with non–small cell lung cancer who are eligible for treatment with the PD-1 inhibitor, cemiplimab (Libtayo).

FDA Grants Fast Track Designation for IK-175 Plus Nivolumab in Bladder Cancer

Also on March 6, the novel aryl hydrocarbon receptor antagonist IK-175 received a fast track designation from the FDA for patients with advanced urothelial carcinoma.

FDA Approves Nelarabine Injection for the Treatment of T-ALL and T-LBL

On March 7, the FDA granted approval to nelarabine injection for the treatment of T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.

FDA Accepts Resubmitted BLA of Remestemcel-L for Pediatric SR-aGVHD

The FDA’s Office of Therapeutic Products accepted the biologics license application resubmission for remestemcel-L for the treatment of children with steroid-refractory acute graft-vs-host disease on March 8.

FDA’s ODAC Votes for Polatuzumab Plus R-CHP for Previously Untreated DLBCL

On March 9, the FDA’s ODAC decided that the benefit/risk profile of polatuzumab vedotin-piiq (Polivy) for injection is favorable for the treatment of previously untreated DLBCL in a vote of 11 to 2.

FDA Type A Meeting Requested to Discuss SGX301 in Early-Stage CTCL

A type A meeting request was submitted to the FDA to discuss the contents of a refusal to file (RTF) letter that was previously issued by the FDA regarding the new drug application for SGX301 (HyBryte) for patients with early-stage cutaneous T-cell lymphoma, also on March 9.

FDA Halts Phase 1 Trial of XMT-2056 for HER2+ Recurrent/Metastatic Solid Tumors

The FDA placed a clinical hold on the phase 1 trial (NCT05514717) of XMT-2056 on March 13, following a report of a patient who experienced a recent grade 5 serious adverse event (SAE) that was deemed to be related to XMT-2056. The SAE and its cause remain under investigation.

FDA Awards Orphan Drug Designation to EP0042 for AML Treatment

The FDA granted an orphan drug designation to EP0042, a dual FLT3 and Aurora kinase inhibitor, on March 14 for patients with acute myeloid leukemia who have acquired resistance to FLT3 inhibitors.

Ripretinib Recognized by FDA and NCCN for Treatment of Second-Line GIST

Also on March 14, the FDA granted a breakthrough therapy designation to ripretinib (Qinlock) for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor who received prior treatment with imatinib (Gleevec) and harbor a KIT exon 11 mutation and co-occurring KIT exon 17 and/or 18 mutations. Ripretinib also was added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines).

FDA Expands Indication of Illuccix for PSMA-Directed Radioligand Therapy

On March 16, the FDA approved a supplementary new drug application for Illuccix, after radiolabeling with gallium (GA)-68 gozetotide injection, for use in select patients with metastatic prostate cancer who are candidates for lutetium Lu 177 vipivotide tetraxetan (Pluvicto)-prostate-specific membrane antigen-directed therapy.

FDA Approves Dabrafenib Plus Trametinib for Pediatric BRAF V600E-Mutant Low-Grade Glioma

The FDA granted approval to dabrafenib (Tafinlar) in combination with trametinib (Mekinist) for the treatment of pediatric patients aged 1 year and older with low-grade glioma that harbor a BRAF V600E mutation and requires systemic therapy, also on March 16.

FDA Accepts IND for GRC 54276 in Advanced Solid Tumors and Lymphomas

On March 16, the FDA also accepted an investigational new drug application for GRC 54276 and the start of a phase 1/2, first-in-human, clinical study of GRC 54276 for the treatment of patients with advanced solid tumors and lymphomas.

FDA Grants FORE8394 Orphan Drug Designation for Primary Brain/CNS Malignancies

The FDA has granted an orphan drug designation on March 20 to FORE8394 for the treatment of patients with primary brain and central nervous system malignancies.

ISB 1442 Lands FDA Orphan Drug Status for R/R Multiple Myeloma

On March 21, the FDA granted an orphan drug designation to ISB 1442, for the treatment of patients with relapsed/refractory multiple myeloma.

FDA Grants FTD to PBP1510 for Unresectable/Metastatic Pancreatic Adenocarcinoma
Also on March 21, the FDA granted a fast track designation to PBP1510 (ulenistamab) for the treatment of patients with unresectable or metastatic pancreatic adenocarcinoma that has relapsed following and/or is refractory to at least 1 prior line therapy.

FDA Approves Retifanlimab for Adults With Advanced Merkel Cell Carcinoma

The FDA granted accelerated approval to retifanlimab (Zynyz) for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma on March 22.

FDA Issues Complete Response Letter to Ruxolitinib XR Tablets for MPNs

The FDA has issued a complete response letter for ruxolitinib (Jakafi) extended-release tablets for once-daily use for the treatment of certain types of myelofibrosis, polycythemia vera, and graft-versus-host disease on March 23.

FDA Grants ODD to Tirabrutinib for Primary Central Nervous System Lymphoma

The FDA granted tirabrutinib, a Bruton's tyrosine kinase inhibitor, an orphan drug designation for the treatment of patients with primary central nervous system lymphoma, also on March 23.

FDA Draft Guidance Supports Randomized Trials for Accelerated Approvals

On March 24, the FDA issued a draft guidance concerning trial design for studies whose purpose is to support accelerated approval of oncology therapeutics, stating its preference for randomized controlled trials over single-arm trials.

FDA Lifts Partial Hold on Phase 1/2 VELA Trial of BLU-222 for Solid Tumors

The FDA lifted the partial clinical hold previously placed on the phase 1/2 VELA trial (NCT05252416) evaluating BLU-222, a selective inhibitor of CDK2, as a treatment for patients with solid tumors on March 28.

FDA Grants Full Approval to Pembrolizumab for MSI-H and dMMR Solid Tumors

On March 29, the FDA granted full approval to pembrolizumab (Keytruda) for the treatment of adult and pediatric patients with unresectable or metastatic MSI-H or dMMR solid tumors, as determined by an FDA-approved test, that have progressed after receiving previous treatment and for those who have no satisfactory alternative treatment options.

Osemitamab Gains FDA Orphan Drug Status for Pancreatic Cancer

On March 29, the FDA also granted an orphan drug designation to osemitamab for the treatment of patients with pancreatic cancer based on findings previously reported at the 2022 International Gastric Cancer Congress which showed osemitamab monotherapy prolonged partial responses in patients with Claudin 18.2 low-expressing pancreatic cancer who progressed after multiple cycles of chemotherapy.

FDA Green Lights FTD of RRx-001 to Mitigate Severe Oral Mucositis in Head and Neck Cancers

Also on March 29, the FDA granted a fast-track designation to RRx-001 for the prevention of severe oral mucositis in chemotherapy- and radiation-treated patients with head and neck cancer.