2025 Orphan Drugs: PDUFA Dates and FDA Approvals

Below is the list of important regulatory dates for all orphan drugs for 2025.   Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. PDUFA DateOrphan DrugIndicationCompanyStatus 1.7.2025. Ryoncil...

A Guide to Italy’s Leading Centers for Rare Diseases

In July of 2024, I visited Denmark and Italy to research their health systems as it pertains to rare diseases. In Denmark, I visited Copenhagen University and met with Oncology professionals to understand their views on rare diseases and rare blood disorders. I also...