by Peter Ciszewski | Jan 1, 2026
Below is the list of important regulatory dates for all orphan drugs for 2026. Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. PDUFA DateOrphan DrugIndicationCompanyStatus 1.5.2026Cablivi (caplacizumab)Acquired...
by Peter Ciszewski | Dec 26, 2025
Current Status of Gene Therapies in Rare Neuromuscular Disorders Alan Beggs, PhD and Julie A Parsons, MD Drs. Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the...
by Peter Ciszewski | Dec 21, 2025
Current Status of Gene Therapy in Lysosomal Storage Disorders Nicola Longo MD, PhD and Mark Roberts, MD In this eight-part podcast series, Drs. Longo and Roberts discuss the current status of gene therapies in lysosomal storage disorders. This content is derived from...
by Peter Ciszewski | Dec 15, 2025
Catching the Clues, Changing the Course of Lysosomal Storage Disorders Professor Yoshikatsu Eto, Dr. Nicole Muschol, Professor Patrício Aguiar, Dr. Robert Hopkin This interactive symposium explores the patient journey across the lysosomal storage disorder (LSD)...
by Peter Ciszewski | Jun 12, 2025
In 2007, the FDA created the Priority Review Voucher (PRV) program to incentivize the development of drugs for rare pediatric and tropical diseases. These PRVs act as a way to skip to the front of the FDA’s line to have a future drug reviewed, which offers...
