2024 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...

2023 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...

2022 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...

Panel Discussion: The High Cost of Rare Diseases  

  Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their organizations’ collective studies showing the direct and...

2021 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...