Tools: Vol 40, No. 4

Approved Drugs

• On April 29, 2025, the US Food and Drug Administration (FDA) approved a ready-todilute formulation of Tepylute^® (thiotepa) (Shorla Oncology) in a 100-mg/10-mL multidose vial for the treatment of patients with breast and ovarian cancer.
• On May 8, the FDA granted accelerated approval to the Avmapki Fakzynja Co-pack (combination of avutometinib and defactinib) (Verastem) for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer who have received prior systemic therapy.
• On May 14, the FDA approved Welireg^® (belzutifan) (Merck) for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma.
• On May 14, the FDA granted accelerated approval to Emrelis^® (telisotuzumab vedotin-tllv) (AbbVie Inc.), a c-Met-directed antibody and microtubule inhibitor conjugate, for adults with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression, as determined by an FDA-approved test, who have received a prior systemic therapy.
• On May 15, the FDA approved Zynyz^® (retifanlimab-dlwr) (Incyte Corporation) with carboplatin and paclitaxel for the first-line treatment of adults with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The FDA also approved retifanlimab-dlwr, as a single agent, for adults with locally recurrent or metastatic SCAC with disease progression on or intolerance to platinum-based chemotherapy.
• On June 3, the FDA approved Nubeqa^® (darolutamide) (Bayer Healthcare Pharmaceuticals) for metastatic castrationsensitive prostate cancer.
• On June 11, the FDA approved Ibtrozi™ (taletrectinib) (Nuvation Bio), a kinase inhibitor, for adults with locally advanced or metastatic ROS1-positive NSCLC.
• On June 11, the FDA approved a new tablet formulation of Brukinsa^® (zanubrutinib) (BeOne Medicines) for all 5 approved indications.
• On June 12, the FDA approved Zusduri® (mitomycin intravesical solution) (UroGen Pharma) for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer.
• On June 12, the FDA approved Keytruda® (pembrolizumab) (Merck) for adults with resectable locally advanced head and neck squamous cell carcinoma whose tumors express PD-L1 as determined by an FDA approved test, as a single agent as neoadjuvant treatment, continued as adjuvant treatment in combination with radiotherapy with or without cisplatin after surgery, and then as a single agent.

Drugs in the News

• The FDA granted fast track designation to emactuzumab (SynOx Therapeutics Limited), a monoclonal antibody that inhibits the CSF-1 receptor, for the treatment of patients with tenosynovial giant cell tumors that are not suitable for or would not benefit from surgery.
• The FDA granted fast track designation to investigational cell therapy iltamiocel (UC Davis Health) for patients experiencing oropharyngeal dysphagia after treatment for head and neck cancer.
• The FDA granted fast track designation to PTX-100 (Prescient Therapeutics), a compound that blocks geranylgeranyl transferase-1, for the treatment of patients with relapsed or refractory mycosis fungoides, the most common subtype of cutaneous T-cell lymphoma.
• The FDA granted orphan drug designation to TPST-1495 (Tempest Therapeutics), a novel dual receptor inhibitor of prostaglandin signaling, for the treatment of patients with familial adenomatous polyposis, a precursor condition to colorectal cancer.
• The FDA granted fast track designation to INX-315 (Incyclix Bio LLC), a CDK2 inhibitor, for the treatment of patients with CCNE1-amplified platinum-resistant/refractory ovarian cancer.
• The FDA granted fast track designation to BI-1808 (BioInvent International), an anti-TNFR2 antibody, for the treatment of patients with relapsed or refractory mycosis fungoides and Sézary syndrome, subtypes of cutaneous T-cell lymphoma.
• The FDA granted fast track designation to ISB 2001 (Ichnos Glenmark Innovation), a trispecific T-cell engager, for the treatment of relapsed or refractory multiple myeloma in patients who have received at least three prior lines of therapy.
• The FDA granted fast track designation to givinostat (Italfarmaco S.p.A.) for the treatment of patients with polycythemia vera, a rare hematologic cancer, for which treatment options are limited.
• The FDA granted fast track designation to ADRX-0706 (Adcentrx Therapeutics) for the treatment of patients with locally advanced or metastatic squamous cell cervical cancer.
• The FDA granted breakthrough therapy designation to ersodetug (Rezolute) for the treatment of hypoglycemia caused by tumor HI.
• The FDA granted orphan drug designation to THE001 (Thermosome) for the treatment of soft tissue sarcomas.
• The FDA granted regenerative medicine advanced therapy designation for B7-H3 (BrainChild Bio), which targets autologous CAR T-cell therapy for the treatment of diffuse intrinsic pontine glioma.
• The FDA granted fast track designation to ZL-1310 (Zai Lab) for the treatment of extensive-stage small cell lung cancer.
• The FDA granted fast track designation to CPO301 (CSPC Pharmaceutical Group Limited), an epidermal growth factor receptor (EGFR) antibody drug conjugate, for the treatment of adult patients with advanced or metastatic non-squamous NSCLC without EGFR mutations or other actionable genomic alterations, with prior disease progression on platinum-based chemotherapy and an anti-PD-(L)1 antibody.
• The FDA granted orphan drug designation to POLB 001 (Poolbeg Pharma), and oral preventative therapy for T-cell engager bispecific antibody-induced cytokine release syndrome.
• The FDA granted fast track designation for EBC-129 (The Experimental Drug Development Centre) for the treatment of pancreatic ductal adenocarcinoma.
• The FDA accepted and granted priority review for Kura Oncology’s new drug application for ziftomenib as a treatment for adult patients with relapsed or refractory acute myeloid leukemia with a nucleophosmin 1 mutation.
• The FDA granted fast track designation to PHST001 (Pheast Therapeutics) for the treatment of patients with advanced platinum-resistant ovarian cancer or in combination with chemotherapy in platinum-sensitive
  ovarian cancer.
• The FDA granted fast track designation to Guardant Health’s Shield multi cancer detection test.
• The FDA granted CHM CDH17 (Chimeric Therapeutics) fast track designation for the treatment of patients with gastroenteropancreatic neuroendocrine tumors.
• The FDA granted breakthrough therapy designation for iopofosine I 131 (Cellectar Biosciences), a cancer targeting agent utilizing a phospholipid ether as a radioconjugate monotherapy, for the treatment of relapsed/ refractory Waldenstrom macroglobulinemia.
• The FDA granted orphan drug designation to MVdeltaC (Oncovita) for the treatment of pleural mesothelioma.
• The FDA granted priority review for Zepzelca^® (lurbinectedin) (Jazz Pharmaceuticals) in combination with Tecentriq^® (atezolizumab) as a first-line maintenance treatment for individuals with extensive-stage small cell lung cancer whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin, and etoposide.
• The FDA granted fast track designation to nadunolimab (Cantargia), an anti-IL1RAP antibody for the treatment of patients with previously untreated metastatic pancreatic ductal adenocarcinoma with high expression levels of IL1RAP in combination with gemcitabine and nab-paclitaxel.
• The FDA granted orphan drug designation to Vasceptor^® (VAS-101) (Vascarta), for the treatment of sickle cell disease

Devices and Assays

• The FDA granted breakthrough device designation to the Ventana^® TROP2 RxDx Assay (EPR20043) (Roche), which uses AI to enhance diagnostic precision in NSCLC by autonomously detecting tumor cells and quantifying TROP2 IHC staining intensity.
• The FDA granted breakthrough device designation to EvoLiver™ (Mursla Bio) for surveillance of hepatocellular carcinoma in patients with high-risk cirrhosis.
• The FDA granted breakthrough device designation for the EnVisio X1™ In-Body Spatial Intelligence™ System (Elucent Medical) for real-time localization and surgical guidance during soft tissue excision, particularly in cancer treatment.
• The FDA approved the Teal Wand™ (Teal Health), an at-home vaginal sample self-collection device for cervical cancer screening.