The Orphan Drug Act provides incentives to sponsors to promote the development of drugs for the prevention, diagnoses, and treatment of rare diseases or conditions that affect fewer than 200,000 individuals in the United States, and for which the sponsor is not...

In September 2021, the Food and Drug Administration (FDA) released its Benefit-Risk Assessment Guidance as part of the Prescription Drug User Fee Amendments (PDUFA) VI Implementation Plan which covers 2018 through 2022.   PDUFA VI focuses on integrating patient...

Antisense oligonucleotide (ASO) drug products target a specific sense mRNA to modulate gene expression in living cells and have been used successfully to treat diseases where the pathogenic mutation has been identified.    For patients with an...

2021 brought more challenges and opportunities than ever before. From the starts and stops of opening workplaces, schools and economies to managing an overflowing email inbox, it can be challenging to keep up with all the information you need. So, we rounded up...

One in 10 Americans is affected by rare diseases—more than half of which are children—and approximately 95% of the more than 7,000 rare diseases have no available treatments. To advance the development of rare disease treatments, the Food and Drug Administration...