On January 16, 2025, the US Food and Drug Administration (FDA) granted approval to Calquence® (acalabrutinib) (AstraZeneca, astrazeneca.com) with bendamustine and rituximab for adults with previously untreated mantle cell lymphoma who are ineligible for autologous hematopoietic stem cell transplantation.
On January 16, the FDA approved Lumakras® (sotorasib) with Vectibix® (panitumumab) (Amgen Inc., www.amgen.com) for adult patients with KRAS G12C-mutated metastatic colorectal cancer, as determined by an FDA-approved test, who have received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy.
On January 17, the FDA approved Datroway® (datopotamab deruxtecan-dlnk) (Daiichi Sankyo, Inc., www.daiichisankyo.com), a Trop-2-directed antibody and topoisomerase inhibitor conjugate, for adult patients with unresectable or metastatic, hormone receptor positive, human epidermal growth factor receptor 2 -negative breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.
On January 21, the FDA approved Grafapex® (treosulfan) (medac GmbH, medac-group.com) with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with acute myeloid leukemia or myelodysplastic syndrome.
On January 27, the FDA approved Enhertu® (trastuzumab deruxtecan) (Daiichi Sankyo, daiichisankyo.com) as the first human epidermal growth factor receptor 2 (HER2)- directed therapy in the United States for adult patients with unresectable or metastatic hormone receptor positive, HER2-low or HER2-ultralow breast cancer.
On February 11, the FDA approved Adcetris® (brentuximab vedotin) (Pfizer, pfizer.com) plus lenalidomide and rituximab for adults with relapsed or refractory large B-cell lymphoma who have undergone at least 2 lines of systemic therapy and are not candidates for autologous hematopoietic stem cell transplantation or CAR-T therapy.
On February 11, the FDA approved Gomekli® (mirdametinib) (Springworks Therapeutics, springworkstx.com) for adults and children aged 2 years and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that cannot be completely resected.
On February 14, the FDA approved Romvimza® (vimseltinib) (Deciphera Pharmaceuticals, LLC, deciphera.com), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor for which surgical resection will potentially cause worsening functional limitation or severe morbidity.
Drugs in the News
The FDA granted fast track designation to AUTX-703 (Auron, aurontx.com) for the treatment of patients with relapsed or refractory acute myelogenous leukemia.
The FDA granted fast track designation for SIGX1094, the world’s first potential targeted therapy for diffuse gastric cancer.
The FDA granted orphan drug designation to OXC-101 (Oxcia, oxcia.com) as a potential treatment option for patients with acute myeloid leukemia.
The FDA granted fast track designation for CUSP06 (OnCusp Therapeutics, oncusptx.com) for the treatment of platinum-resistant ovarian cancer.
The FDA granted orphan drug designation for OPN-6602 (Opna Bio, opnabio.com), an oral EP300/CBP bromodomain inhibitor, for multiple myeloma.
The FDA granted a second fast track designation for IBI363 (Chimerix, chimerix.com) a bispecific antibody fusion protein, for the treatment of unresectable, locally advanced, or metastatic squamous non-small cell lung cancer.
The FDA has accepted for review the resubmission of the biologics license application for linvoseltamab (Regeneron Pharmaceuticals Inc, regeneron.com) for the treatment of relapsed/refractory multiple myeloma.
The FDA granted regenerative medicine advanced therapy designation for sterically optimized CAR-T NXC-201 (Immix Biopharma, immixbio.com), for relapsed/refractory AL amyloidosis.
The FDA granted fast track designation to amezalpat (TPST-1120) (Tempest Therapeutics, tempesttx.com) for the treatment of patients with hepatocellular carcinoma.
The FDA granted fast track designation for eRapa (Biodexa Pharmaceuticals, biodexapharma.com) a proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis.
The FDA granted orphan drug designation to Ac-SSO110 (satoreotide) (Ariceum Therapeutics, ariceum-therapeutics.com) for the treatment of patients with small cell lung cancer.
The FDA granted regenerative medicine advanced therapy designation for Vigil® (gemogenovatucel-t) (Gradalis Inc, gradalisinc.com) as a maintenance treatment for women with newly diagnosed, advanced Stage IIIb/IV ovarian cancer who are homologous recombination proficient, have a high clonal tumor mutation burden and who are in complete response after debulking surgery and frontline platinum-based doublet chemotherapy.
The FDA granted orphan drug designation for MB-105 (March Bio Sciences, marchbiosciences.com) a first-in-class CD5 CAR-T cell therapy for T-Cell Lymphoma.
The FDA granted rare pediatric disease and orphan drug designations for RBM39 degrader ST-01156 (Seed Therapeutics, seedtheraputics.com).
The FDA granted priority review to Welireg® (belzutifan) (Merck, merck.com) for the treatment of patients with advanced pheochromocytoma and paraganglioma.
The FDA granted fast track designation for Cu-64 SAR-bisPSMA (Clarity Pharmaceuticals, claritypharmaceuticals.com) in biochemical recurrence of prostate cancer.
The FDA granted orphan drug designation for ZL-1310 (Zai Lab, zailaboratory.com) a potential highly active first-in-class DLL3 antibody-drug conjugate for the treatment of small cell lung cancer.
The FDA granted breakthrough therapy designation for letetresgene autoleucel (lete-cel) (Adaptimmune, adaptimmune.com) for the treatment of patients myxoid/round cell liposarcoma who have received prior anthracy[1]cline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen.
The FDA granted orphan drug designation to ELC-100 (Elicera, elicera.com) for the treatment of pancreatic neuroendocrine tumors.
The FDA granted fast track designation for LOAd703 (Lokon Pharma, lokonpharma.com) for the treatment of pancreatic cancer.
The FDA granted additional fast track designation for XMT-1660 (Mersana Therapeutics, mersana.com) for the treatment of advanced or metastatic breast cancer in patients with HER2 low or HER2-negative disease, including triple-negative breast cancer, who have received a prior topoisomerase-1 inhibitor ADC.
The FDA granted orphan drug designation to R289 (Rigel, rigel.com) for the treatment of myelodysplastic syndromes.
The FDA granted fast track designation for Azenosertib (Zentalis, zentalis.com) for the treatment of patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who are positive via Cyclin E1 immunohistochemistry for protein levels.
The FDA granted fast track designation for LYT-200 (PureTech, puretechhealth.com) an anti-galectin-9 monoclonal antibody, for the treatment of acute myeloid leukemia.
The FDA granted fast track designation for BBO-8520 (BridgeBio Oncology Therapeutics, bridgebiooncology.com) an investigational oral therapy for the treatment of adult patients with previously treated KRASG12C-mutated metastatic non-small cell lung cancer (NSCLSC).
The FDA granted fast track designation to STAR0602 (Marengo, marengotx.com) for treatment of unresectable, locally advanced, or metastatic colorectal cancers with high tumor mutational burden.
The FDA granted fast track designation for VGT-309 (abenacianine) for injection (Vergent Bioscience, vergentbio.com) as an adjunct for the intraoperative visualization of primary lung cancer, other pulmonary lesions, cancer containing lymph nodes, and positive surgical margins in patients undergoing surgery for known or suspected cancer in the lung.
The FDA granted priority review to sunvozertinib (Dizal Pharma, dizalpharma.com) an oral epidermal growth factor receptor inhibitor for the treatment of locally advanced or metastatic NSCLC.
The FDA granted breakthrough therapy designation to GSK’227 (GSK, gsk.com) for the treatment of adult patients with relapsed or refractory osteosarcoma who have progressed on at least two prior lines of therapy.
The FDA granted orphan drug designation to amezalpat (Tempest Therapeutics, tempesttx.com) to treat patients with hepatocellular carcinoma.
Devices and Assays
The FDA granted breakthrough device designation for DCISionRT Test®(Prelude DX, preludedx.com) for DCIS breast cancer patients.
The FDA has granted breakthrough device designation for ACR-368 OncoSignature (Acrivion Therapeutics, acrivion.com) assay for endometrial cancer.
The FDA granted breakthrough device designation to HyperVIEW (Lumitron Technologies, lumitronxrays.com) a cancer detection X-Ray system.
The FDA granted approval of an investigational device exemption to expand the pilot trial of Alpha DaRT® (Alpha Tau, alphatau.com) to 30 patients with pancreatic cancer in 2 cohorts, with locally advanced or metastatic disease.
The FDA granted approval for the PATHWAY anti-HER-2 (Roche, roche.com) rabbit monoclonal primary antibody assay as a companion diagnostic device to identify patients with HER2-ultralow breast cancer for treatment with Enhertu®. This assay was previously approved to identify patients with HER2-low breast cancer for treatment with Enhertu.
