Business · 30/09/2021

FDA Releases Final Guidance on Gene Therapy Products for Orphan-Drug Designation

The US Food and Drug Administration (FDA) has released a final guidance document titled 'Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations' to help industry stakeholders determine the sameness of human gene therapy products for orphan-drug designation and exclusivity, with the goal of developing treatments for rare diseases.