Tools: Vol. 41, No. 4

Approved Drugs

• On May 1, 2026, the FDA approved Veppanu (vepdegestrant) (Arvinas), a heterobifunctional protein degrader, for adults with estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy.
• On May 8, the FDA approved Bizengri® (zenocutuzumab-zbco) (Partner Therapeutics) for adults with advanced, unresectable, or metastatic cholangiocarcinoma harboring a neuregulin 1 gene fusion with disease progression on or after prior systemic therapy.
• On May 13, the FDA granted accelerated approval to Beqalzi™ (sonrotoclax) (BeOne Medicines), a BCL-2 inhibitor, for adults with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton's tyrosine kinase inhibitor.
• On May 13, the FDA approved Inqovi® (decitabine and cedazuridine) (Taiho Oncology) with venetoclax for the treatment of newly diagnosed acute myeloid leukemia in adults 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy.
• On May 15, the FDA approved Tecentriq® (atezolizumab) (Genentech) and Tecentriq Hybreza® (atezolizumab and hyaluronidase-tqjs) (Genentech) as adjuvant treatments for adults with muscle invasive bladder cancer after cystectomy who have circulating tumor DNA molecular residual disease as determined by an FDA-authorized test.
• On May 15, the FDA approved Enhertu® (fam-trastuzumab deruxtecan-nxki) (Daiichi Sankyo) for two separate indications in adults with HER2-positive early-stage breast cancer. The first indication is for Enhertu followed by a taxane, trastuzumab, and pertuzumab, for the neoadjuvant treatment of adult patients with HER2-positive (IHC 3+ or ISH+) stage II or III breast cancer, as determined by an FDA-authorized test. The second indication is for Enhertu for the adjuvant treatment of adult patients with HER2-positive (IHC 3+ or ISH+) breast cancer who have residual invasive disease following neoadjuvant treatment with trastuzumab (with or without pertuzumab) and taxane-based treatment.
• On May 22, the FDA approved Datroway® (datopotamab deruxtecan-dlnk) (Daiichi Sankyo) for adult patients with unresectable or metastatic triple-negative breast cancer who are not candidates for PD-1/PD-L1 inhibitor therapy.
• On May 27, the FDA approved Decnupaz™ (pivekimab sunirine-pvzy) (AbbVie), a CD123-directed antibody and alkylating agent conjugate, for adults with blastic plasmacytoid dendritic cell neoplasm.
• On May 28, the FDA approved Imfinzi (durvalumab) (AstraZeneca) in combination with Bacillus Calmette-Guerin (BCG) for the treatment of adult patients with BCG-naïve, high-risk non-muscle invasive bladder cancer.
• On June 12, the FDA approved Welireg® (belzutifan) (Merck) in combination with Keytruda® (pembrolizumab) (Merck) or Keytruda Qlex™ (pembrolizumab and berahyaluronidase alfa-pmph) (Merck) for the adjuvant treatment of adults with renal cell carcinoma with a clear cell component at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
• On June 12, the FDA approved Truqap (capivasertib) (AstraZeneca) in combination with abiraterone and prednisone for adults with metastatic androgen pathway modulation-naïve or -sensitive prostate cancer (previously referred to as metastatic hormone-sensitive prostate cancer) that is PTEN-deficient as detected by an FDA-authorized test.
• On June 24, the FDA approved Ibrance® (palbociclib) (Pfizer) in combination with trastuzumab, with or without pertuzumab, and endocrine therapy for the maintenance treatment of adults with HR-positive, HER2-positive locally advanced or metastatic breast cancer following induction treatment.
• On June 24, the FDA approved Trodelvy® (sacituzumab govitecan-hziy) (Gilead) for two indications in adults with triple-negative breast cancer.
• On June 30, the FDA approved TREGZI™ (Orca-T®) (Orca Bio), an allogeneic regulatory T cell-based immunotherapy with hematopoietic stem and progenitor cell and T cells-vldq, for use in matched donor hematopoietic stem cell transplantation with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease-free survival, in the treatment of adults with hematological malignancies.

Drugs in the News

• The FDA granted fast track designation to DOC1021 (Diakonos Oncology), a first-in-class, patient-derived double-loaded dendritic cell investigational therapy, for the treatment of unresectable or metastatic cutaneous melanoma.
• The FDA granted regenerative medicine advanced therapy designation to RZ-001 (Rznomics), an investigational candidate for the treatment of hepatocellular carcinoma.
• The FDA granted fast track designation to RPTR-1-201 (Repertoire® Immune Medicines), a T cell receptor bispecific immune medicine, for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic triple-negative breast cancer after progression on, or intolerance to, available standard therapies.
• The FDA granted fast track designation to SIR2501 (Sironax), a SARM1 inhibitor, for chemotherapy-induced peripheral neuropathy.
• The FDA granted fast track designation to zocilurtatug pelitecan (Zai Lab), a Delta-like ligand 3-targeting antibody-drug conjugate, for the treatment of extrapulmonary neuroendocrine carcinomas following progression after standard first-line therapy.
• The FDA granted breakthrough therapy designation to emiltatug ledadotin (Servier), an investigational antibody drug conjugate directed against B7-H4, for the treatment of patients with locally advanced, recurrent, or metastatic adenoid cystic carcinoma with solid histology or high-grade transformation.
• The FDA granted fast track designation to Suplexa (Alloplex Biotherapeutics) for the treatment of patients with colorectal cancer of the MSI-H phenotype.
• The FDA granted Hyrnuo® (sevabertinib) (Bayer) priority review status for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer whose tumors have HER2 (ERBB2) tyrosine kinase domain-activating mutations in patients with no prior therapy.
• The FDA granted orphan drug designation to CLN-049 (Cullinan Therapeutics), a novel, investigational FLT3xCD3 T cell engager, for the treatment of relapsed/refractory acute myeloid leukemia.
• The FDA granted rare pediatric disease designation and orphan drug designation to FL118 (Canget BioTekpharma), a novel anticancer drug candidate, for the treatment of osteosarcoma.
• The FDA granted priority review to the new drug application for neladalkib (Nuvalent).
• The FDA granted orphan drug designation to EO2463 (Enterome) for the treatment of patients with indolent non-Hodgkin lymphoma in the low-tumor-burden, "watch-and-wait" setting.
• The FDA granted fast track designation to STX-0712 (Solu Therapeutics), an investigational therapy in development for the treatment of relapsed or refractory chronic myelomonocytic leukemia.
• The FDA granted orphan drug designation to oxaliplatin (RenovoRx) for the treatment of pancreatic cancer.
• The FDA granted priority review to the new drug application for bezuclastinib (Cogent Biosciences) in combination with sunitinib for patients with gastrointestinal stromal tumors who have received prior treatment with imatinib.
• The FDA granted fast track designation to NOUS-209 (Nouscom) for the prevention of Lynch Syndrome (LS)-associated cancers in LS carriers with genetically confirmed mismatch repair mutations.
• The FDA granted orphan drug designation to SOT106 (SOTIO Biotech), an antibody-drug conjugate targeting leucine-rich repeat-containing 15, for the treatment of osteosarcoma.
• The FDA granted fast track designation to TER-2013 (Terremoto Biosciences), an AKT1-selective inhibitor, in patients with locally advanced, unresectable, or metastatic HR+/HER2- breast cancer harboring one or more AKT/PI3K/PTEN alterations following progression on at least one endocrine-based therapy and CDK4/6 inhibitor for advanced disease.
• The FDA granted fast track designation to VS-7375 (Verastem Oncology), an oral investigational selective KRAS G12D (ON/OFF) inhibitor, for the treatment of adult patients with KRAS G12D-mutated unresectable locally advanced or metastatic non-small cell lung cancer who have received platinum-based chemotherapy and an anti-PD-(L)1 antibody either concurrently or sequentially.
• The FDA granted fast track designation to azer-cel (azercabtagene zapreleucel) (Imugene Limited) for two indications: relapsed or refractory chronic lymphocytic leukaemia/small lymphocytic lymphoma and relapsed or refractory marginal zone lymphoma.
• The FDA granted rare pediatric disease designation to opaganib (RedHill Biopharma) for treatment of neuroblastoma.
• The FDA granted regenerative medicine advanced therapy designation to lasmecabtagene timgedleucel (lasme-cel) (Cellectis), its CD22-targeting allogeneic CAR-T cell therapy product candidate, for the treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukemia.
• The FDA accepted the supplemental biologics license application filing and granted priority review for adjuvant Tecentriq® (atezolizumab) (Roche) and Tecentriq Hybreza® (atezolizumab and hyaluronidase-tqjs) in combination with chemotherapy in stage III deficient DNA mismatch repair or microsatellite instability-high colon cancer.
• The FDA granted fast track designation to ofirnoflast (HT-6184) (Halia Therapeutics) for the treatment of adult patients with lower-risk myelodysplastic syndromes.
• The FDA granted fast track designation to givastomig (ABL111) (NovaBridge Biosciences) in combination with nivolumab and chemotherapy for the treatment of patients with previously untreated HER2-negative advanced or metastatic gastroesophageal adenocarcinomas whose tumors are both Claudin 18.2 and PD-L1 positive.

Devices and Assays

• The FDA granted breakthrough device designation to Vesta Bladder Risk Stratify Dx (Valar Labs), an AI-powered digital pathology prognostic test for bladder cancer.
• The FDA granted breakthrough device designation to SPOT-MAS 10 (Gene Solutions), a multi-omic blood test designed to detect cancer-associated signals.
• The FDA granted breakthrough device designation to Merlin CP-GEP¹ (SkylineDx), a clinicopathologic gene expression profile test designed to support risk assessment and clinical decision-making in patients with early-stage cutaneous melanoma.