2026 Orphan Drugs: PDUFA Dates and FDA Approvals ++++++

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. Below is the list of important regulatory dates for all orphan drugs for 2026. Accelerated Approval Granted to Loargys (pegzilarginase) for the Treatment of ARG1 Deficiency https://checkrare.com/accelerated-approval-granted-to-loargys-pegzilarginase-for-the-treatment-of-arg1-deficiency/ The US FDA has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of patients ages 2 years and older with arginase 1 deficiency (ARG1-D). FDA Approves 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). FDA Approved Darzalex Faspro Combination Therapy for Patients With Transplant Ineligible Newly Diagnosed Multiple Myeloma https://checkrare.com/fda-approved-darzalex-faspro-combination-therapy-for-patients-with-transplant-ineligible-newly-diagnosed-multiple-myeloma/ The US FDA has approved Darzalex Faspro (daratumumab and hyaluronidase) in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for the treatment of adults with newly diagnosed multiple myeloma (MM) who are ineligible for autologous stem cell transplant (ASCT). Expanded Indication for Cablivi to Patients Ages 12 Years and Older With aTTP https://checkrare.com/fda-expands-cablivi-caplacizumab-indication-to-patients-ages-12-years-and-older-with-attp/ The U.S. FDA has approved Cablivi (caplacizumab) for the treatment of pediatric patients ages 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. FDA Approves Aqvesme (Mitapivat) for Treating Anemia in Alpha- and Beta-Thalassemia https://checkrare.com/fda-approves-aqvesme-mitapivat-for-treating-anemia-in-alpha-and-beta-thalassemia/ The U.S. FDA has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.