FDA's July Roundup: Approvals, Fast Tracks, and Designations in Oncology
Key Insights
The FDA approved FoundationOne Liquid CDx (search) as a companion diagnostic for niraparib and abiraterone in BRCA-mutated metastatic castration-resistant prostate cancer (search).
Several therapies received fast track designations, including OBX-115 for advanced melanoma (search) and ADI-270 for metastatic clear cell renal cell carcinoma (search).
Biologics license applications were accepted for remestemcel-L in pediatric steroid-refractory acute graft-vs-host disease (search) and tabelecleucel for Epstein-Barr virus-positive posttransplant lymphoproliferative disease (search).
July 2024 saw a flurry of activity at the FDA, with multiple approvals, fast track designations, and key regulatory decisions impacting cancer treatment. These actions span diagnostic tools, novel therapies, and trial design considerations, reflecting the agency's ongoing efforts to advance oncology care.
Companion Diagnostic Approval for Prostate Cancer
The FDA approved FoundationOne Liquid CDx (search) as a companion diagnostic for niraparib (Zejula) and abiraterone acetate (Akeega) in patients with BRCA-mutated metastatic castration-resistant prostate cancer (search) (mCRPC). This approval allows for more precise identification of patients who may benefit from this targeted therapy combination.
Fast Track Designations
Several therapies received fast track designations, which are intended to expedite the development and review of drugs that treat serious conditions and fill unmet medical needs. These include:
- OBX-115: An innovative therapy for advanced melanoma that has relapsed or is refractory to PD-1/PD-L1 inhibitors.
- 225Ac-FL-020: For the treatment of mCRPC.
- ADI-270: For treating metastatic or advanced clear cell renal cell carcinoma (ccRCC) previously treated with an immune checkpoint inhibitor and a VEGF inhibitor.
- DSP-5336: For relapsed/refractory AML with a KMT2A rearrangement or NPM1 mutation.
- DOC1021: A novel dendritic cell vaccine for the treatment of pancreatic ductal adenocarcinoma.
- Ozuriftamab vedotin (CAB-ROR2-ADC; BA3021): For the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC).
Regulatory Submissions and Designations
The FDA accepted biologics license applications (BLAs) for remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (search) (SR-aGVHD) and tabelecleucel (tab-cel; Ebvallo) for the treatment of Epstein-Barr virus-positive posttransplant lymphoproliferative disease (search). Additionally, SLS009 (formerly GFH009) was granted a rare pediatric disease designation for the treatment of pediatric patients with AML, and 7MW3711 received an orphan drug designation for small cell lung cancer.
Clinical Trial Developments
The FDA enforced a partial clinical hold on a phase 1 trial of seclidemstat (SP-2577) with azacitidine in myelodysplastic syndrome (search) (MDS) or chronic myelomonocytic leukemia (search) (CMML). An IND application for SenoVax was filed with the FDA for the potential treatment of patients with NSCLC. The Prescription Drug User Fee Act target action date for the new drug application for revumenib (SNDX-5613) was extended by the FDA to December 26, 2024.
ODAC Recommendation on NSCLC Trial Designs
On July 25, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously (11-0) to recommend that new trial design proposals for perioperative regimens for resectable non-small cell lung cancer (search) (NSCLC) include a comprehensive assessment of each treatment phase's contribution. This decision aims to improve the evaluation of treatment efficacy and safety in NSCLC, potentially leading to improved patient outcomes.
Other Approvals and Actions
- Guardant Shield, a blood test to detect cancer in the colon or rectum, was approved.
- The IND application for ZW191, a novel folate receptor alpha-targeting ADC, was cleared.
- Asciminib (Scemblix) was granted priority review for the treatment of newly diagnosed Philadelphia chromosome-positive, chronic phase chronic myeloid leukemia (CML).
- The IND application to begin a phase 1 study for COM503, a potential first-in-class treatment for solid tumors, was cleared.
- Daratumumab and hyaluronidase-fihj regimen (Darzalex Faspro) in combination with bortezomib (Velcade), lenalidomide (Revlimid), and dexamethasone (D-VRd) was approved for newly diagnosed multiple myeloma patients eligible for autologous stem cell transplant.
- The IND application for UB-VV111 for the potential treatment of hematologic malignancies was cleared.
- VCN-01 was granted rare pediatric disease designation for retinoblastoma.