FDA NEWS & CLINICAL PERSPECTIVES ++++ +++++

FDA NEWS 2025 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2025-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 2025. FDA Approves Ziftomenib for Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia https://checkrare.com/fda-approves-ziftomenib-for-relapsed-refractory-npm1-mutated-acute-myeloid-leukemia/ The U.S. FDA has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation. Darzalex Faspro for Patients With High-Risk Smoldering Multiple Myeloma https://checkrare.com/fda-approves-darzalex-faspro-for-patients-with-high-risk-smoldering-multiple-myeloma/ The U.S. FDA has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). FDA Approves Revumenib for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia https://checkrare.com/fda-approves-revumenib-for-the-treatment-of-relapsed-refractory-acute-myeloid-leukemia/ The U.S. FDA has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). CLINICAL PERSPECTIVES Long-Term Data From the AQUARIUS Study of Avacopan in Patients With GPA and MPA https://checkrare.com/long-term-data-from-the-aquarius-study-of-avacopan-in-patients-with-gpa-and-mpa/ Naomi Patel, MD, MPH, Rheumatologist at Massachusetts General Hospital, discusses long-term data from the AQUARIUS study of avacopan in patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). Hemolytic Disease of the Fetus and Newborn: Outcomes of Intrauterine Transfusion and Patient Experiences https://checkrare.com/hemolytic-disease-of-the-fetus-and-newborn-outcomes-of-intrauterine-transfusion-and-patient-experiences/ May Lee Tjoa, PhD, Senior Global Medical Affairs Leader: Nipocalimab and Maternal-Fetal Immunology at Johnson & Johnson, discusses data on hemolytic disease of the fetus and newborn (HDFN) from the 2025 ISUOG World Congress on Ultrasound in Obstetrics and Gynecology. Rilzabrutinib Approval for Adult Patients With Immune Thrombocytopenia https://checkrare.com/rilzabrutinib-approval-for-adult-patients-with-immune-thrombocytopenia/ Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP). ISUOG World Congress 2025: Hemolytic Disease of the Fetus and Newborn https://checkrare.com/isuog-world-congress-2025-hemolytic-disease-of-the-fetus-and-newborn/ Jannine Williams, Compound Development Team Leader at Johnson & Johnson, discusses key takeaways from studies on hemolytic disease of the fetus and newborn (HDFN) presented at ISUOG World Congress 2025. Plans for Phase 2/3 Clinical Trial of Bexmarilimab Plus Standard of Care in Patients With Myelodysplastic Syndromes https://checkrare.com/plans-for-phase-2-3-clinical-trial-of-bexmarilimab-plus-standard-of-care-in-patients-with-myelodysplastic-syndromes/ Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, discusses plans for a phase 2/3 clinical trial testing bexmarilimab plus standard of care in patients with myelodysplastic syndromes (MDS).