Tools: Vol. 41, No.3

Approved Drugs

• On March 5, 2026, the FDA approved Tecvayli® (teclistamab) (Janssen Biotech) in combination with Darzalex Faspro® (daratumumab hyaluronidase-fihj) for adult patients with relapsed or refractory multiple myeloma who have received at least 1 prior line of therapy including a proteasome inhibitor and an immunomodulatory agent.
• On March 20, the FDA approved Opdivo® (nivolumab) (Bristol Myers Squibb) with doxorubicin, vinblastine, and dacarbazine for adult and pediatric patients 12 years and older with previously untreated stage III or IV classical Hodgkin lymphoma.
• On March 25, the FDA approved Lifyorli™ (relacorilant) (Corcept Therapeutics), a glucocorticoid receptor antagonist, in combination with nab-paclitaxel for the treatment of adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received 1 to 3 prior systemic treatment regimens, at least 1 of which included bevacizumab.

Drugs in the News

• The FDA granted priority review to rusfertide (Takeda and Protagonist Therapeutics), an investigational, first-in-class hepcidin mimetic peptide therapeutic for the treatment of adults with polycythemia vera.
• The FDA accepted and granted priority review to the supplemental biologics license application for Enhertu (trastuzumab deruxtecan) (AstraZeneca and Daiichi Sankyo) for the treatment of adult patients with HER2-positive breast cancer who have residual invasive disease after neoadjuvant HER2-targeted treatment.
• The FDA granted orphan drug designation to zolucatetide (Parabilis Medicines), an investigational Helicon™ peptide and a direct inhibitor of β-catenin:TCF interaction, for the treatment of desmoid tumors.
• The FDA granted orphan drug designation to AMXT 1501 (Aminex Therapeutics) in combination with difluoromethylornithine for the treatment of malignant glioma, including diffuse intrinsic pontine glioma.
• The FDA granted orphan drug designation to irinotecan (CRISM Therapeutics) for the treatment of malignant glioma.
• The FDA granted fast track designation to FG001 (FluoGuide) as an intraoperative imaging agent to guide surgical resection of high-grade glioma.
• The FDA granted fast track designation to small molecule MYB mRNA degrader REM-422 (Remix Therapeutics) for the treatment of patients with recurrent, metastatic or unresectable adenoid cystic carcinoma whose tumors express MYB transcripts containing a poison exon.
• The FDA granted orphan drug designation to RCT1213 (ReCerise Therapeutics), an antibody targeting TM4SF5, for the treatment of hepatocellular carcinoma.
• The FDA granted fast track designation to TRI-611 (TRIANA Biomedicines), an investigational molecular glue degrader therapy, for the treatment of anaplastic lymphoma kinase–positive non-small cell lung cancer.
• The FDA granted breakthrough therapy designation to WU-CART-007 (soficabtagene geleucel), a cell-based immunotherapy developed by Washington University School of Medicine, for the treatment of T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.
• The FDA granted fast track designation to ZW191 (Zymeworks), an antibody-drug conjugate targeting folate receptor-α, for the treatment of patients with advanced or metastatic platinum-resistant ovarian cancer.
• The FDA granted priority review to the new drug application for lirafugratinib (Elevar Therapeutics), an investigational therapy for the treatment of patients with cholangiocarcinoma with FGFR2 fusions or rearrangements who have received prior therapy.
• The FDA granted regenerative medicine advanced therapy designation to CB-011 (Caribou Biosciences), an allogeneic anti-BCMA CAR T-cell therapy, for relapsed or refractory multiple myeloma.
• The FDA granted fast track designation to PAS-004 (Pasithea Therapeutics) for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas causing significant morbidity.
• The FDA granted fast track designation to A2B543 (A2 Biotherapeutics), an autologous chimeric antigen receptor (CAR) T-cell therapy designed for the treatment of germline heterozygous HLA-A02 adults with recurrent unresectable, locally advanced, or metastatic solid tumors that express mesothelin and have lost HLA-A02 expression.
• The FDA granted breakthrough therapy designation to plixorafenib (FORE Biotherapeutics) for the treatment of adult patients with BRAF V600E-mutated high-grade glioma.
• The FDA granted fast track designation to CTIM-76 (Context Therapeutics), a CLDN6 x CD3 T cell engaging bispecific antibody, for the treatment of platinum-resistant ovarian cancer in patients that have received all standard of care therapies.
• The FDA granted fast track designation to SIM0505 (NextCure), an investigational antibody-drug conjugate comprised of an antibody that targets Cadherin-6 and a proprietary topoisomerase 1 inhibitor payload, for the treatment of platinum-resistant ovarian cancer.
• The FDA granted orphan drug designation to Tovecimig (CTX-009/ABL001) (ABL Bio), a bispecific antibody, for the treatment of bile duct cancer.
• The FDA granted orphan drug designation to REYOBIQ™ (rhenium Re186 obisbemeda) (Plus Therapeutics) for the treatment of pediatric malignant gliomas.
• The FDA granted fast track designation to CT-P71 (Celltrion), an antibody-drug conjugate, for the treatment of patients with locally advanced or metastatic urothelial carcinoma.
• The FDA accepted and granted priority review to Daiichi Sankyo and Merck’s biologics license application for I-DXd (ifinatamab deruxtecan) for the treatment of adult patients with extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy.
• The FDA granted fast track designation to Daretabart (hu1418K322A) (Renaissance Pharma), a novel anti-GD2 monoclonal antibody in development for the treatment of high-risk neuroblastoma.
• The FDA granted orphan drug designation to eftilagimod alfa (Immutep) for the treatment of soft tissue sarcoma.
• The FDA granted fast track designation to OPN-6602 (Opna Bio), a dual EP300/CBP inhibitor, for the treatment of patients with relapsed/refractory multiple myeloma who have received at least 4 prior lines of therapy.
• The FDA accepted for priority review a supplemental biologics license application for perioperative PADCEV™ (enfortumab vedotin-ejfv) (Astellas Pharma), a Nectin-4 directed antibody-drug conjugate, in combination with the PD-1 inhibitor Keytruda® (pembrolizumab) or Keytruda QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph) (Merck) as treatment for patients with muscle-invasive bladder cancer.
• The FDA granted orphan drug designation to ODM-212 (Orion Pharma) for the treatment of mesothelioma.
• The FDA granted fast track designation to lunresertib (Debio2513) (Debiopharm), a PKMYT1 inhibitor, in combination with zedoresertib (Debio 0123), a WEE1 inhibitor.
• The FDA granted fast track designation to BBO-11818 (BridgeBio Oncology Therapeutics) for the treatment of adult patients with advanced KRAS-mutant pancreatic ductal adenocarcinoma.
• The FDA granted breakthrough therapy designation to TERN-701 (Terns Pharmaceuticals), a novel, oral allosteric BCR::ABL1 inhibitor, for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase without the T315I mutation, previously treated with 2 or more tyrosine kinase inhibitors.
• The FDA granted fast track designation to ImmunoBody® iSCIB1+ (Scancell) for the treatment of advanced melanoma.
• The FDA granted regenerative medicine advanced therapy designation to Orca-Q® (Orca Bio), a second-generation investigational allogeneic T-cell immunotherapy, for the treatment of high-risk hematologic malignancies.
• The FDA granted priority review to a supplemental biologics license application for TEVIMBRA® (tislelizumab) (BeOne) in combination with ZIIHERA® (zanidatamab) and chemotherapy for the first-line treatment of unresectable locally advanced/metastatic HER2-positive gastric, gastroesophageal junction, or esophageal adenocarcinoma. The FDA has also granted breakthrough therapy designation to the regimen of ZIIHERA in combination with fluoropyrimidine- and platinum-containing chemotherapy, with and without TEVIMBRA, in this indication.

Devices and Assays

• The FDA granted breakthrough device designation to Cognita Chest X-Ray™ (Mosaic Clinical Technologies™), an industry-first generative vision-language model designed to assist radiologists in the interpretation of chest X-rays.
• The FDA granted breakthrough device designation to TOBY’s non-invasive, urine-based multi-cancer early detection test.
• The FDA granted breakthrough device designation to CatCHimera (TCM Biotech), a liquid biopsy MRD platform for hepatocellular carcinoma.
• The FDA granted breakthrough device designation to the AidaBREAST® test (PreludeDx), an early-stage invasive breast cancer assay.