Drug discovery today
Non-oncology orphan drug development: Productivity and probability of success.
Samantha Parker, Jida El Hajjar, Anneliene H Jonker, Susan R Kahn, Persefoni Kritikou, Christina Kyriakopoulou, Anthony Haight
Published: 202510.1016/j.drudis.2025.104568
Abstract
There are >6000 rare diseases (RDs), affecting >300 million people worldwide. Despite increases in orphan drug designations (ODDs), approved treatments remain scarce. This study analyzes the outcomes of non-oncology ODs designated by the FDA and Euro…
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