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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. Kresladi (Marnetegragene Autotemcel) for Children with LAD-1 https://checkrare.com/accelerated-fda-approval-of-ex-vivo-gene-therapy-for-children-with-lad-1/ The US FDA has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-1 (LAD-1). Avalayah (Tividenofusp Alfa) for MPS II https://checkrare.com/accelerated-approval-granted-to-mps-ii-enzyme-replacement-therapy-that-crosses-blood-brain-barrier/ The US FDA has granted accelerated approval to Avlayah (tividenofusp alfa) for the treatment of neurologic manifestations of Hunter syndrome (MPS II). 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). Lynavoy (Linerixibat) for Cholestatic Pruritus in Primary Biliary Cholangitis https://checkrare.com/fda-approves-lynavoy-linerixibat-for-cholestatic-pruritus-in-primary-biliary-cholangitis/ The US FDA has approved Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with primary biliary cholangitis (PBC). Lomitapide for Pediatric Patients With Homozygous Familial Hypercholesterolemia https://checkrare.com/fda-expands-indication-of-lomitapide-to-pediatric-patients-with-homozygous-familial-hypercholesterolemia/ The US FDA has approved Juxtapid (lomitapide) capsules for the treatment of pediatric patients ages 2 years and older with homozygous familial hypercholesterolemia (HoFH). Wellcovorin (Leucovorin) for Patients With Cerebral Folate Deficiency https://checkrare.com/fda-approves-wellcovorin-leucovorin-for-patients-with-cerebral-folate-deficiency/ The US FDA has approved Wellcovorin (leucovorin calcium) tablets for the treatment of adult and pediatric patients with cerebral folate deficiency (CFD) with confirmed variants in the folate receptor 1 gene (CFD-FOLR1). Potential Treatment for ROSAH Syndrome Accepted into FDA’s Rare Disease Evidence Principles Process https://checkrare.com/potential-treatment-for-rosah-syndrome-accepted-into-fdas-rare-disease-evidence-principles-process/ The US FDA has accepted DF-003 into the FDA Rare Disease Evidence Principles Process (RDEP) for the potential treatment of ROSAH syndrome.