Rare Disease Focus: Oncology / Rare Cancers +++++ ++++ ++++

FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs.  Tecartus for Treatment of Adults With Mantle Cell Lymphoma https://checkrare.com/full-approval-granted-to-tecartus-for-treatment-of-adults-with-mantle-cell-lymphoma/ The US FDA has approved Tecartus (brexucabtagene autoleucel) for the treatment of adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL). FDA Approves Nivolumab With AVD for Patients With Classical Hodgkin Lymphoma https://checkrare.com/fda-approves-nivolumab-with-avd-for-patients-with-classical-hodgkin-lymphoma/ The US FDA has approved Opdivo (nivolumab) with doxorubicin, vinblastine, and dacarbazine (AVD) for adult and pediatric patients 12 years and older with previously untreated, Stage 3 or 4 classical Hodgkin lymphoma (cHL). Tecvayli for Multiple Myeloma Under the National Priority Voucher Program https://checkrare.com/fda-grants-approval-to-multiple-myeloma-combination-therapy-under-the-national-priority-voucher-program/ The US FDA has approved Tecvayli (teclistamab-cqyv) plus Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adults with relapsed or refractory multiple myeloma. Venetoclax Combination Therapy for Adults With Chronic Lymphocytic Leukemia https://checkrare.com/fda-approves-venetoclax-combination-therapy-for-adults-with-chronic-lymphocytic-leukemia/ The US FDA has approved Venclexta (venetoclax) in combination with acalabrutinib therapy for untreated adult patients with chronic lymphocytic leukemia (CLL). CLINICAL PERSPECTIVES Arms Wide Open Childhood Cancer Foundation and CureFest https://checkrare.com/arms-wide-open-childhood-cancer-foundation-and-curefest/ Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses her organization and the CureFest event. FDA’s Plausible Mechanism Framework and its Effect on Rare Disease Therapy Development https://checkrare.com/fdas-plausible-mechanism-framework-and-its-effect-on-rare-disease-therapy-development/ Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. How The FDA’s Plausible Mechanism Framework Is Accelerating Approval for Osteosarcoma Immunotherapy https://checkrare.com/how-the-fdas-plausible-mechanism-framework-is-accelerating-approval-for-osteosarcoma-immunotherapy/ Paul Romness, CEO of OS Therapies, discusses the Plausible Mechanism Framework draft guidance and how it will affect the approval process of OST-HER2 LM for the treatment of osteosarcoma. MajesTEC-9 Clinical Trial of Teclistamab in Patients With Multiple Myeloma https://checkrare.com/majestec-9-clinical-trial-of-teclistamab-in-patients-with-multiple-myeloma/ Roberto Mina, MD, Associate Professor at Winship Cancer Institute of Emory University, discusses the Majestec-9 clinical trial of Tecvayli (teclistamab) in patients with multiple myeloma (MM). Using Artificial Intelligence to Analyze Castleman Disease Histopathology https://checkrare.com/using-artificial-intelligence-to-analyze-castleman-disease-histopathology/ Robert S. Ohgami, MD, PhD, Professor of Pathology, University of Utah, and Founding Vice President and Chief Medical Director, ARUP Institute for Research and Innovation, discusses the utilization of artificial intelligence (AI) to analyze Castleman disease (CD) histopathology. Elritercept’s Effect on Transfusion Independence in Patients With Myelodysplastic Syndromes https://checkrare.com/elritercepts-effect-on-transfusion-independence-in-patients-with-myelodysplastic-syndromes/ Lynette Chee, PhD, Hematologist at The Royal Melbourne Hospital/ Peter MacCallum Cancer Centre, discusses elritercept’s effect on transfusion independence (TI) in patients with myelodysplastic syndromes (MDS). 52-Week Results From the VERIFY Clinical Trial of Rusfertide in Patients With Polycythemia Vera https://checkrare.com/52-week-results-from-the-verify-clinical-trial-of-rusfertide-in-patients-with-polycythemia-vera/ Andrew T. Kuykendall, MD, VERIFY Lead Investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses 52-week results from the VERIFY clinical trial testing rusfertide in patients with polycythemia vera (PV). Bleximenib Combination Therapy for Patients With Acute Myeloid Leukemia https://checkrare.com/bleximenib-combination-therapy-for-patients-with-acute-myeloid-leukemia/ Hartmut Döhner, MD, Professor of Medicine and Medical Director of the Department of Hematology and Oncology, Ulm University, Germany, discusses bleximenib combination therapy for treatment of patients with acute myeloid leukemia (AML).