How many novel drugs will be approved by the US Food and Drug Administration for rare (aka "orphan") diseases in 2024?
Mini
5
Ṁ815
Jan 1
1.1%
Under 12
3%
Between 12 and 19
75%
Between 20 and 27
15%
Between 28 and 35
6%
More than 35

Over the past decade, the percentage of novel drug approvals for rare diseases (diseases that affect fewer than 200,000 people in the US) has increased (FDA - Rare Disease Day 2023, Pharmaceutical Technology). In 2022, the FDA approved 20 novel drugs for rare diseases (FDA - New Drug Therapy Approvals 2022).

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