News|Articles|October 1, 2025
September 2025 FDA Roundup: This Month's Regulatory Recap
Author(s)Targeted Oncology Staff
Fact checked by: Sabrina Serani
Listen
0:00 / 0:00
Key Takeaways
- Zongertinib and olomorasib received breakthrough therapy designations for specific NSCLC mutations, enhancing treatment options for these patient populations.
- Denosumab biosimilars Bildyos and Bilprevda were approved for osteoporosis and cancer-related bone loss, expanding therapeutic choices.
September sees pivotal FDA approvals and designations, advancing innovative cancer treatments and reshaping oncology care for various malignancies.
September was a dynamic month for oncology regulation, marked by several significant decisions from the US FDA that will shape the treatment landscape for multiple cancer types. Here are the FDA's most impactful oncology regulatory happenings from the past month.
Zongertinib (Hernexeos), a highly selective oral HER2 TKI, was granted breakthrough therapy designation (BTD) by the FDA for the first-line treatment of adult patients with unresectable or metastatic nonsquamous non–small cell lung cancer (NSCLC) harboring activating HER2 mutations on September 3.
On September 3, the FDA granted approval for Bildyos and Bilprevda, 2 biosimilars to denosumab, a monoclonal antibody targeting the receptor activator of nuclear factor kappa-B ligand (RANKL), for the treatment of osteoporosis and cancer-related bone loss.
On September 4, the FDA granted breakthrough therapy designation to olomorasib in combination with pembrolizumab (Keytruda) for the treatment of unresectable advanced or metastatic non–small cell lung cancer (NSCLC) harboring a KRAS G12C mutation and PD-L1 expression ≥50%.
On September 5, the FDA granted fast track designation to CER-1236, an investigational cellular immunotherapy, for the treatment of acute myeloid leukemia (AML).
On September 8, the FDA granted orphan drug designation to BA-101, an investigational agent for the treatment of glioblastoma, marking an important step forward in identifying novel therapies in this hard-to-treat disease.
On September 9, the FDA approved the intravesical gemcitabine delivery system Inlexzo, previously known as TAR-200, for the treatment of certain types of bladder cancer following unsuccessful Bacillus Calmette-Guérin therapy or for patients who refuse/are ineligible for radical cystectomy.
On September 10, the FDA approved selumetinib (Koselugo) for the treatment of pediatric patients 1 year of age and older with neurofibromatosis type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN).
On September 10, the FDA granted fast track designation to GLSI-100 for patients with HER2-positive breast cancer with an HLA-A*02 genotype.
Also on September 10, the FDA accepted the new drug application for relacorilant in combination with nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer. This submission is supported by data from the pivotal phase 3 ROSELLA trial (NCT05257408) and earlier phase 2 studies, highlighting a potential new therapeutic option in a population with limited effective treatments.
On September 15, the FDA granted breakthrough therapy designation to the novel antibody-drug conjugate (ADC) raludotatug deruxtecan (R-DXd; DS-6000a) for the treatment of patients with platinum-resistant epithelial ovarian, primary peritoneal, or fallopian tube cancers expressing CDH6 who received prior treatment with bevacizumab (Avastin).
On September 16, the FDA granted orphan drug designation to ligufalimab (AK117) for the treatment of AML. Ligufalimab is a next-generation humanized IgG4 monoclonal antibody targeting CD47. Its design prevents red blood cell agglutination and has demonstrated improved safety and efficacy compared with other CD47-targeting agents.
On September 16, the FDA has granted fast track designation to the ADC CRB-701 for the treatment of recurrent or metastatic head and neck squamous cell carcinoma previously treated with platinum-based chemotherapy and an anti–PD-(L)1 agent.
On September 17, the FDA approved Bosaya and Aukelso (denosumab-kyqq) as biosimilars for Prolia and Xgeva, respectively, for the treatment of cancer- and treatment-related bone loss.
On September 18, the FDA granted fast track designation to MNV-201 for the treatment of myelodysplastic syndromes. MNV-201 also received fast track and rare pediatric disease designation for the treatment of Pearson Syndrome, an ultrarare mitochondrial disorder affecting pediatric patients.
On September 19, the FDA approved the biologics license application for the subcutaneous (SC) administration of pembrolizumab (Keytruda Qlex) formulated with berahyaluronidase alfa (MK-3475A) across all previously approved solid tumor indications for pembrolizumab, including metastatic NSCLC.
The FDA has approved the CytoCell KMT2A Breakapart FISH Probe Kit PDx as a companion diagnostic (CDx) to accompany revumenib (Revuforj), a first-in-class Menin inhibitor for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients 1 year and older.
On September 24, the FDA granted priority review to the supplemental biologics license application of fam-trastuzumab deruxtecan-nxki (T-DXd; Enhertu) for the first-line treatment of patients with unresectable or metastatic HER2-positive breast cancer.
On September 25, the FDA approved imlunestrant (Inluriyo) for the treatment of adult patients with ER-positive, HER2–, ESR1-mutated advanced or metastatic breast cancer whose disease has progressed after at least 1 line of endocrine therapy.
In conjuction with the approval of imlunestrant, the FDA approved the Guardant 360 CDx as a companion diagnostic to identify ESR1 mutation in patients with advanced breast cancer.
On September 25, the FDA removed the risk evaluation and mitigation strategies (REMS) program for the thyroid cancer medication vandetanib (Caprelsa).
Related Content
Advertisement
