Approved Drugs

• On November 15, 2024, the US Food and Drug Administration (FDA) approved Revuforj® (revumenib) (Syndax Pharmaceuticals, syndax.com), a menin inhibitor, for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients 1 year and older.
• On November 19, the FDA approved updated drug labeling for Fludarabine Phosphate Injection (fludarabine phosphate) (Sandoz, sandoz.com) under Project Renewal, an Oncology Center of Excellence initiative aimed at updating labeling information for certain older oncology drugs to ensure information is clinically meaningful and scientifically up to date.
• On November 20, the FDA granted accelerated approval to Ziihera® (zanidatamab-hrii) (Jazz Pharmaceuticals, jazzpharma.com), a bispecific HER2-directed antibody, for previously treated, unresectable or metastatic, HER2-positive biliary tract cancer as detected by an FDA-approved test.
• On December 4, the FDA granted accelerated approval to Bizengri® (zenocutuzumab-zbco) (Merus NV, merus.nl), a HER2-HER3–specific antibody, for adults with the following: advanced, unresectable, or metastatic non-small cell lung cancer (NSCLC) harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy; or advanced, unresectable, or metastatic pancreatic adenocarcinoma harboring a NRG1 gene fusion with disease progression on or after prior systemic therapy.
• On December 4, the FDA approved Imfinzi® (durvalumab) (AstraZeneca, astrazeneca.com) for adults with limited-stage small cell lung cancer whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy.
• On December 13, the FDA approved Unloxcyt® (cosibelimab-ipdl) (Checkpoint Therapeutics, checkpointtx.com), a programmed death ligand-1 blocking antibody, for adults with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation.
• On December 18, the FDA approved Ensacove® (ensartinib) (Xcovery Holdings, xcovery.com) for adult patients with anaplastic lymphoma kinase (ALK)-positive, locally advanced or metastatic NSCLC who have not previously received an ALK-inhibitor.
• On December 18, the FDA approved Ryoncil® (remestemcel-L-rknd) (Mesoblast, mesoblast.com), an allogeneic bone marrow– derived mesenchymal stromal cell therapy, for the treatment of steroid-refractory acute graft versus host disease in pediatric patients 2 months of age and older.
• On December 20, the FDA granted accelerated approval to Braftovi® (encorafenib) (Pfizer, pfizer.com) with cetuximab and modified fluorouracil, leucovorin calcium, and oxaliplatin for patients with metastatic colorectal cancer with a BRAF V600E mutation as detected by an FDA-approved test.
• On December 27, the FDA approved Opdivo Qvantig® (nivolumab and hyaluronidase-nvhy) (Bristol Myers Squibb, bms.com) for subcutaneous injection across approved adult, solid tumor Opdivo® (nivolumab) indications as monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy® (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib.

Drugs in the News

• The FDA granted rare pediatric disease designation to elraglusib (Actuate Therapeutics, actuatetherapeutics.com), a novel GSK-3 beta inhibitor, to treat Ewing sarcomas.
• The FDA granted both orphan drug and rare pediatric disease designations to SRN-101 (Siren Biotechnology, sirenbiotechnology.com), an immuno-gene candidate, for the treatment of high-grade gliomas.
• The FDA granted fast-track designation to ALE.P02 (Alentis Therapeutics, alentis.ch), an investigational antibody-drug conjugate, to treat advanced or metastatic CLDN1-positive squamous cancers irrespective of the organ of origin.
• The FDA granted breakthrough therapy designation to RP1 (vusolimogene oderparepvec) (Replimune, replimune.com) in combination with Opdivo to treat patients with advanced melanoma who have previously received anti-PD1 therapy.
• The FDA granted orphan drug designation to LBL-024 (Leads Biolabs, leadsbiolabs.com) a bispecific antibody, to treat neuroendocrine tumors.
• The FDA granted fast-track designation to LBS-007 (Lin BioScience, linbioscience.com), a non-ATP cell cycle inhibitor, to treat acute myeloid leukemia.
• The FDA granted breakthrough therapy designation to sac-TMT (sacituzumab tirumotecan) (Merck, merck.com) for the treatment of patients with advanced or metastatic nonsquamous NSCLC with epidermal growth factor receptor mutations whose disease progressed on or after tyrosine kinase inhibitor and platinum-based chemotherapy.
• The FDA granted fast track designation for investigational drug candidate LP-184 (Lantern Pharma, lanternpharma.com) for the treatment of triple-negative breast cancer.
• The FDA granted fast track designation to CRB-701 (Corbus Pharmaceuticals Holdings, corbuspharma.com) for the treatment of relapsed or refractory metastatic cervical cancer.
• The FDA granted fast track designation to R289 (Rigel Pharmaceuticals, rigel.com) for the treatment of patients with previously treated, transfusion-dependent, lower-risk myelodysplastic syndrome.
• The FDA granted orphan drug designation to PLT012 (Pilatus Biosciences, pilatusbio.com) for treating liver and intrahepatic bile duct cancer.
• The FDA granted breakthrough therapy designation to SER-155 (Seres Therapeutics, serestherapeutics.com), the company’s lead investigational program, for the reduction of bloodstream infections in adults undergoing allogeneic hematopoietic stem cell transplant to treat hematological malignancies.
• The FDA granted orphan drug designation to azeliragon (Cantex Pharmaceuticals, cantex.com) for the treatment of brain metastasis from breast cancer.
• The FDA granted priority review to AstraZeneca’s (astrazeneca.com) supplemental biologics license application for Imfinzi® (durvalumab) for the treatment of patients with muscle-invasive bladder cancer.
• The FDA granted fast track designation for the oncolytic adenovirus-delivered transforming growth factor beta inhibitor, AdAPT-001 (EpicentRx, epicentrx.com), plus the anti–PD-1 agent nivolumab or the anti–PD-L1 agent atezolizumab, to treat recurrent or refractory advanced or metastatic soft tissue sarcoma with disease progression after at least 1 prior line of therapy.
• The FDA granted fast track designation to PT217 (Phanes Therapeutics, phanesthera.com) for the treatment of patients with metastatic de novo or treatment-emergent neuroendocrine prostate cancer.
• The FDA granted fast track designation to IMM-1-104 (Immuneering Corporation, immuneering.com) as a treatment for patients with unresectable or metastatic NRAS-mutant melanoma who have progressed on or are intolerant to PD-1/PD-L1–based immune checkpoint inhibitors.
• The FDA granted breakthrough therapy designation to Jemperli® (dostarlimab-gxly) (GSK, gsk.com) for the treatment of patients with locally advanced mismatch repair– deficient/microsatellite instability–high rectal cancer.
• The FDA has designated Thio® (MAIA Biotechnology, maiabiotech.com) for the treatment of pediatric-type diffuse high-grade gliomas as a drug for a rare pediatric disease.
• The FDA granted breakthrough therapy designation to Trodelvy® (sacituzumab govitecan-hziy) (Gilead Sciences, gilead.com) for the treatment of adult patients with extensive-stage small cell lung cancer whose disease has progressed on or after platinumbased chemotherapy.
• The FDA granted fast track designation to NX-5948 (Nurix Therapeutics, nurixtx.com), a highly selective degrader of Bruton tyrosine kinase (BTK), for the treatment of adult patients with relapsed or refractory Waldenstrom macroglobulinemia after at least 2 lines of therapy, including a BTK inhibitor.
• The FDA granted orphan drug designation to VGT-1849A (Vanda Pharmaceuticals, vandapharma.com), a selective antisense oligonucleotide-based JAK2 inhibitor, for the treatment of polycythemia vera.
• The FDA accepted for review a new drug application under the accelerated approval pathway for avutometinib (Verastem Oncology, verastem.com), an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent, low-grade, serous ovarian cancer, who received at least 1 prior systemic therapy and have a KRAS mutation.