FDA May 2024 Roundup: A Month of Progress in Cancer Treatment and Detection

May 2024 saw advancements in various areas, including a significant number of FDA approvals, designations, and clearances related to cancer treatment and detection.

The FDA approved several novel cancer therapies, including a subcutaneous formulation of nivolumab (Opdivo) for multiple solid tumors, selpercatinib (Retevmo) for the treatment of pediatric patients aged 2 and older with RET-altered thyroid cancer or solid tumors, and ColoSense™, a noninvasive stool test for colorectal cancer screening.

Promising drugs for specific cancers like NVL-655 for ALK-positive non-small cell lung cancer (NSCLC) and inavolisib (GDC-0077) for PIK3CA-mutated breast cancer received breakthrough therapy designations from the FDA, and several agents gained fast track designations from the FDA, such as SNB-101 for small cell lung cancer and RAG-01, marking the first small activating RNA drug to receive this status.

The FDA also approved protocols for phase 3 trials aiming to test new treatments for patients with metastatic castration-resistant prostate cancer and pancreatic cancer, and the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (ODAC) met on May 22, 2024, to discuss the impact of the FDA Reauthorization Act (FDARA) on pediatric cancer drugs.

Here is a look back on all the FDA happenings from the month of May 2024.

FDA Accepts BLA for Subcutaneous Nivolumab Across Various Solid Tumors

On May 6, 2024, the biologics license application (BLA) of the subcutaneous formulation of nivolumab coformulated with human hyaluronidase (rHuPH20) was accepted by the FDA for all previously approved adult solid tumor indications of nivolumab as a monotherapy, monotherapy maintenance following completion of the combination therapy with nivolumab plus ipilimumab (Yervoy), or in combination with chemotherapy or cabozantinib (Cabometyx).

FDA Clears IND for EIS-12656, a Novel Therapy for Multiple Solid Tumors

The FDA cleared the investigational new drug application for EIS-12656 for the treatment of patients with various solid tumors, also on May 6, 2024.

FDA to Consider Zenocutuzumab in NRG1+ NSCLC and Pancreatic Cancer

The FDA also granted priority review to the BLA of zenocutuzumab (MCLA-128) on May 6, 2024, in patients with neuregulin 1 fusion-positive NSCLC and pancreatic ductal adenocarcinoma.

FDA Oks ColoSense, a Stool Test for Early Colorectal Cancer Detection

The FDA approved ColoSense™, a noninvasive stool test for colorectal cancer screening in average-risk adults 45 and older, on May 6, 2024.

9MW2821 Gains FDA Orphan Drug Designation in Esophageal Cancer

On May 6, 2024, 9MW2821, an antibody-drug conjugate targeting Nectin-4, also received orphan drug designation for treating esophageal cancer.

FDA Clears IND of BTX-9341 in HR+/HER2- Breast Cancer

The FDA cleared the investigational new drug application for BTX-9341 on May 7, 2024, for the treatment of hormone receptor (HR)-positive/HER2-negative breast cancer.

FDA Approves IND of Epstein-Barr mRNA Cancer Vaccine WGc-043

The FDA approved the investigational new drug application of WGc-043, an Epstein-Barr virus-related mRNA cancer vaccine, on May 10, 2024.

FDA Grants Fast Track Designation to SNB-101 in SCLC

A fast track designation was granted to SNB-101 by the FDA on May 10, 2024, for small cell lung cancer treatment.

FDA Clears IND Application for SynKIR-310 Trial in R/R B-NHL

The FDA cleared an investigational new drug application for SynKIR-310 for the treatment of patients with relapsed/refractory B-cell non-Hodgkin lymphoma on May 14, 2024.

FDA Greenlights HPV Self-Collection Solution for Cervical Cancer Screening

The FDA granted approval to the use of self-collected samples for human papillomavirus (HPV) testing when cervical samples cannot be obtained on May 15, 2024.

NVL-655 Earns FDA Breakthrough Therapy Designation in ALK-Positive NSCLC

On May 16, 2024, the FDA granted a breakthrough therapy designation to NVL-655, a novel brain-penetrant ALK-selective tyrosine kinase inhibitor (TKI) for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have received 2 or more ALK TKIs.

FDA Withdraws Infigratinib Approval for Cholangiocarcinoma

The FDA approval for infigratinib (Truseltiq) to treat cholangiocarcinoma was withdrawn on May 16, 2024.

Inavolisib Regimen Wins FDA Breakthrough Therapy Designation for PIK3CA+ Breast Cancer

The FDA granted a breakthrough therapy designation to inavolisib plus palbociclib (Ibrance) and fulvestrant for the treatment of HR-positive, HER2-negative, PIK3CA-mutated breast cancer on May 21, 2024.

RAG-01 Gains FDA Fast Track Designation in NMIBC

A fast track designation was granted to RAG-01 by the FDA on May 21, 2024, making it the first small activating RNA drug worldwide to receive this designation.

FDARA’s Impact on Pediatric Cancer Drugs: Insights From May 2024 ODAC Meeting

On May 22, 2024, the Pediatric Oncology Subcommittee of the ODAC meeting convened to discuss the impact of amendments made by Section 504 of the FDARA to Section 505B of the Food, Drug, and Cosmetic Act, which was passed in 2017.

FDA Oks Phase 3 Trial of LAE201 for the Treatment of mCRPC

The FDA approved the phase 3 trial protocol to test a combination therapy (LAE201) for metastatic castration-resistant prostate cancer that has progressed on standard treatments on May 23, 2024.

VCN-01 Plus Chemo Wins FDA Fast Track Designation in Pancreatic Cancer

Also on May 23, 2024, the FDA granted a fast track designation to VCN-01 given in combination with chemotherapy for the treatment of patients with metastatic pancreatic adenocarcinoma.

FDA Advisory Committee Recommends Shield Blood Test Approval for CRC Screening

The FDA’s Medical Devices Advisory Committee also gave the green light to the Guardant Health’s Shield blood test for colorectal cancer screening in average-risk adults aged 45 and older on May 23, 2024.

Avutometinib/Defactinib Seeks FDA Approval for Ovarian Cancer via Rolling NDA

On May 24, 2024, a rolling submission of a new drug application to the FDA was initiated, seeking accelerated approval of avutometinib (previously VS-6766), a RAF/MEK clamp, given in combination with defactinib, a selective FAK inhibitor, for the treatment of adult patients with recurrent KRAS-mutated low-grade serous ovarian cancer previously treated with at least 1 prior systemic therapy.

Inavolisib Regimen Wins FDA Priority Review for PIK3CA Breast Cancer

The FDA granted priority review to the new drug application of inavolisib plus palbociclib and fulvestrant on May 29, 2024, for the treatment of HR-positive, HER2-negative, PIK3CA-mutated breast cancer.

Zanidatamab BLA Receives FDA Priority Review for HER2+ Biliary Tract Cancer

The FDA accepted and granted priority review of the BLA for zanidatamab (ZW25) for the treatment of patients with previously treated, HER2-positive, unresectable, locally advanced or metastatic biliary tract cancer, also on May 29, 2024.

Selpercatinib Wins FDA Accelerated Approval for RET-Altered Tumors in Pediatric Patients

On May 29, 2029, the FDA also granted accelerated approval to selpercatinib for the treatment of patients aged 2 and older with RET-altered thyroid cancer or solid tumors.

FDA Grants AFM24 Plus Atezolizumab Fast Track Designation in Advanced NSCLC

Also on May 29, 2024, the FDA granted a fast track designation to AFM24 combined with atezolizumab (Tecentriq) for the treatment of patients with advanced and/or metastatic NSCLC without activating EGFR mutations after progression on PD-1/PD-L1 therapy and platinum-based chemotherapy.

Pembrolizumab/Chemo sBLA Receives FDA Priority Review in Advanced Mesothelioma

The FDA granted priority review to a supplemental biologics license application of pembrolizumab (Keytruda) plus chemotherapy in unresectable or metastatic malignant pleural mesothelioma on May 29, 2024.

FDA Grants RMAT Designation to TSC-100 and TSC-101 for Hematologic Malignancies

The FDA granted regenerative medicine advanced therapy designation to TSC-100 and TSC-101 for the treatment of patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation with reduced intensity conditioning on May 29, 2024.

FDA Greenlights First-in-Human Trial of ACTM-838 for Advanced Solid Tumors

On May 30, 2024, the FDA approved an investigational new drug application that initiates a phase 1 trial of ACTM-838 for the treatment of solid tumors.

FDA Approves Liso-Cel in Mantle Cell Lymphoma

The FDA approved the chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel (liso-cel; Breyanzi) on May 30, 2024, for the treatment of adult patients with relapsed or refractory mantle cell lymphoma after treatment with 2 or more lines of therapy, including a Bruton tyrosine kinase inhibitor.

AGuIX Gains FDA Fast Track Status in Malignant Gliomas and Glioblastoma

The FDA granted a fast track designation to AGuIX for treating malignant gliomas and glioblastoma on May 30, 2024.

FDA Acknowledges BLA of Zolbetuximab in HER2-, CLDN18.2+ Gastric/GEJ cancer

Also on May 30, 2024, the FDA has acknowledged the resubmitted BLA for zolbetuximab (Vyloy) as a first-line treatment for locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are claudin 18.2-positive.