FDA's July 2024 Roundup: Breakthroughs and Milestones in Cancer Treatment

July 2024 proved to be a landmark month for the FDA, with significant approvals, fast track designations, and key submissions. Notably, the FDA approved the FoundationOne Liquid CDx as a companion diagnostic for niraparib (Zejula) and abiraterone acetate (Akeega) in patients with BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC), as well as Guardant Shield, a blood test to detect cancer in the colon or rectum.

The FDA granted 6 fast track designations, including one to OBX-115, an innovative therapy for advanced melanoma that has relapsed or is refractory to PD-1/PD-L1 inhibitors. A regenerative medicine advanced therapy (RMAT) designation was granted to AIC100 for recurrent anaplastic thyroid cancer and is expected to expedite the development of this novel chimeric antigen receptor (CAR) T-cell therapy.

Two biologics license applications were also accepted by the FDA, including 1 for remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (SR-aGVHD) and 1 for tabelecleucel (tab-cel; Ebvallo) for the treatment of Epstein-Barr virus-positive posttransplant lymphoproliferative disease.

On July 25, the FDA’s Oncologic Drugs Advisory Committee (ODAC) met and voted to require comprehensive phase assessments for new trial designs in perioperative regimens for non–small cell lung cancer (NSCLC). The goal of this decision is to improve the evaluation of treatment efficacy and safety in NSCLC, with the hopes of potentially improving outcomes for patients who are undergoing surgery.

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

​​FDA Approves FoundationOne Liquid CDx for Niraparib/Abiraterone in Aggressive Prostate Cancer

On July 1, 2024, FoundationOne Liquid CDx became an FDA-approved companion device for niraparib and abiraterone acetate in deleterious or suspected deleterious BRCA-mutated mCRPC.

DNA Molecule : © ktsdesign - stock.adobe.com

Cosibelimab BLA Resubmitted to FDA for Metastatic cSCC

The biologics license application of cosibelimab (formerly CK-301) for metastatic or locally advanced cutaneous squamous cell carcinoma (cSCC) was resubmitted to the FDA on July 2, 2024.

FDA Fast Tracks 225Ac-FL-020 in mCRPC

A fast track designation was granted to ²²⁵Ac-FL-020 on July 2, 2024, for the treatment of mCRPC.

FDA Clears IND of Intravenous JCXH-211 in Advanced Solid Tumors

The FDA granted clearance to an investigational new drug (IND) application of intravenous JCXH-211 in patients with advanced solid tumors on July 8, 2024.

ADI-270 Gains FDA Fast Track Status in Metastatic/Advanced ccRCC

Also on July 8, 2024, a fast track designation was given to ADI-270 from the FDA for treating metastatic or advanced clear cell renal cell carcinoma (ccRCC) previously treated with an immune checkpoint inhibitor and a VEGF inhibitor.

BLA for Remestemcel-L to Treat Pediatric SR-aGVHD Resubmitted to FDA

The biologics license application for remestemcel-L for the treatment of pediatric patients with SR-aGVHD was resubmitted to the FDA on July 9, 2024.

FDA Wraps Up End of Phase 2 Meeting for AML Combination Therapy

The end of phase 2 meeting between the FDA and Moleculin Biotech, Inc. for the phase 1b/2 trial (NCT05319587) of the combination of annamycin and cytarabine in patients with acute myeloid leukemia (AML) was completed on July 10, 2024.

FDA Receives 505(b)(2) NDA Seeking Approval for Lutetium Lu 177 Dotatate

On July 9, 2024, a 505(b)(2) new drug application seeking the approval of Curium’s formulation of lutetium Lu 177 dotatate injection for treating somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors was submitted to the FDA.

FDA Fast Tracks Innovative TIL Therapy OBX-115 in Advanced Melanoma

The FDA granted a fast track designation to OBX-115 on July 9, 2024, for the treatment of metastatic melanoma that is refractory to or relapsed following treatment with PD-1/PD-L1–based immune checkpoint inhibitors.

FDA Enforces Partial Hold on Trial of Seclidemstat in MDS/CMML

A partial clinical hold was placed on a phase 1 trial (NCT04734990) of the combination of seclidemstat (SP-2577) with azacitidine in myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML), also on July 9, 2024.

FDA Receives IND Submission for Phase 1/2 Trial of Silmitasertib in Solid Tumors

An IND application for a phase 1/2 trial of silmitasertib (CX-4945) plus chemotherapy in pediatric and young adult patients with relapsed/refractory solid tumors was submitted to the FDA on July 11, 2024.

Medical illustration featuring cancer cells from a microscopic to macroscopic view, illuminated by neon lights to enhance visual clarity and focus: © Fay Melronna - stock.adobe.com

FDA Fast Tracks DSP-5336 in NPM1/KMT2A+ Acute Leukemia

On July 15, 2024, the FDA granted DSP-5336 a fast track designation, supported by updated findings from an ongoing phase 1/2 study in relapsed/refractory AML with a KMT2A rearrangement, also known as mixed lineage leukemia rearrangement or NPM1 mutation.

FDA Fast Tracks Novel Dendritic Cell Vaccine for Pancreatic Cancer

Also on July 15, 2024, the FDA granted a fast track designation for DOC1021 for the treatment of pancreatic ductal adenocarcinoma.

FDA Grants Rare Pediatric Disease Designation to SLS009 in Pediatric AML

The FDA granted SLS009 (formerly GFH009) a rare pediatric disease designation for the treatment of pediatric patients with AML on July 16, 2024.

FDA Grants Orphan Drug Designation to Novel B7-H3-Targeting ADC in SCLC

Also on July 16, 2024, the FDA granted 7MW3711 an orphan drug designation for the treatment of small cell lung cancer.

FDA Accepts Application of Tab-Cel, First-in-Class Therapy for EBV+ PTLD

On July 17, 2024, the FDA accepted and granted priority review to the biologics license application of tab-cel for the treatment of Epstein-Barr virus-positive posttransplant lymphoproliferative disease.

Phase 3 Trials of Botensilimab/Balstilimab Move Forward, Despite FDA's Approval Setback

During an end of phase 2 meeting between the FDA and Agenus, the FDA advised against filing for accelerated approval of botensilimab (AGEN1181) combined with balstilimab (AGEN2034) for the treatment of relapsed/refractory microsatellite stable metastatic colorectal cancer without liver metastases as the objective response rates observed in the phase 2 trial (NCT05608044) of the combination may not translate to a survival benefit. This was on July 18, 2024.

FDA Clears IND of ZW191, Novel Folate Receptor Alpha-Targeting ADC

The FDA cleared the investigational new drug application for ZW191, a novel antibody-drug conjugate (ADC) on July 22, 2024.

New CAR T Granted FDA RMAT Designation for Anaplastic Thyroid Cancer

The FDA granted AIC100 RMAT designation on July 23, 2024, for the treatment of recurrent anaplastic thyroid cancer.

FDA Approves Second Dose of CAR T-Cell Therapy in Ovarian Cancer Trial

An individual patient IND application that allows for the second dose of a CAR T-cell therapy was approved by the FDA, also on July 23, 2024.

FDA Accepts Remestemcel-L BLA Resubmission for Acute GVHD

On July 23, 2024, the FDA also accepted the resubmitted biologics license application of remestemcel-L for the treatment of pediatric patients with SR-aGVHD.

FDA Fast Tracks Ozuriftamab Vedotin in Recurrent/Metastatic HNSCC

A fast track designation was granted to ozuriftamab vedotin (CAB-ROR2-ADC; BA3021) from the FDA for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) on July 23, 2024.

FDA Accepts NDA of TLX007-CDx in Prostate Cancer

The FDA accepted the filing of a new drug application (NDA) for TLX007-CDx on July 24, 2024.

Tumor microenvironment concept with cancer cells: © catalin - stock.adobe.com

FDA ODAC Votes Unanimously to Mandate Phase Assessments in NSCLC Perioperative Regimens

In an 11 to 0 vote, the FDA’s ODAC agreed on July 25, 2024, that the FDA should mandate that new trial design proposals for perioperative regimens for resectable NSCLC include an adequate trial assessment of the contribution of each treatment phase.

New CAR T Therapy for ALL Earns FDA Orphan and Rare Pediatric Drug Designations

The FDA granted orphan drug and rare pediatric disease designations to UCART22 for the treatment of acute lymphoblastic leukemia on July 25, 2024.

SenoVax Investigational New Drug Application Filed With the FDA in NSCLC

An IND application for SenoVax was filed with the FDA for the potential treatment of patients with NSCLC on July 26, 2024.

FDA Approves Shield Blood Test for Colorectal Cancer Screening

On July 19, 2024, the FDA approved Guardant Shield, a blood test to detect cancer in the colon or rectum.

Asciminib Gains FDA Priority Review in Newly Diagnosed CML

Also on July 29, 2024, the FDA granted priority review to the application of asciminib (Scemblix) for the treatment of newly diagnosed Philadelphia chromosome-positive, chronic phase chronic myeloid leukemia (CML).

FDA Clears COM503 IND in Solid Tumors, Phase 1 Trial Anticipated

On July 29, 2024, the FDA also cleared the IND application to begin a phase 1 study for COM503, a potential first-in-class treatment for solid tumors.

FDA Extends PDUFA Date for Revumenib NDA for KMT2A-Rearranged Acute Leukemia

The Prescription Drug User Fee Act target action date for the new drug application for revumenib (SNDX-5613) was extended by the FDA on July 29, 2024, to December 26, 2024.

Promising Pancreatic Cancer Treatment Receives FDA Orphan Drug Designation

On July 29, 2024, the FDA also granted an orphan drug designation to avutometinib (VS-6766) plus defactinib (VS-6063) for the treatment of pancreatic cancer.

FDA Approves Daratumumab Plus VRd in Newly Diagnosed Multiple Myeloma

On July 30, 2024, daratumumab and hyaluronidase-fihj regimen (Darzalex Faspro) in combination with bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (D-VRd) was approved by the FDA for the treatment of patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant.

FDA Clears IND Application of UB-VV111 in Hematologic Malignancies

The FDA cleared the IND application for UB-VV111 for the potential treatment of hematologic malignancies on July 31, 2024.

FDA Grants VCN-01 Rare Pediatric Disease Designation in Retinoblastoma

The FDA also granted rare pediatric disease designation to VCN-01 for the treatment of retinoblastoma on July 31, 2024.