Tuesday, November 5, 2024

Navigating the Orphan Medicinal Product Designation: Evidence Requirements for Gene Therapies in Europe

Palomo, G.M. et al., Navigating the Orphan Medicinal Product Designation: Evidence Requirements for Gene Therapies in Europe. Molecular Therapy, Volume 0, Issue 0. DOI: 10.1016/j.ymthe.2024.10.015 

To provide insight into regulatory decision-making at the time of granting initial orphan designation by the Committee for Orphan Medicinal Products, we have conducted a retrospective analysis for viral vector-mediated gene therapies in rare non-oncological conditions with respect to the data provided to support the criteria to be met in successful applications.