FDA orphan drug designations for lysosomal storage disorders - a cross-sectional analysis.

ABSTRACT:

Purpose

To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat lysosomal storage disorders (LSDs).

Methods

Assessment of the drug pipeline through analysis of the FDA database for orphan drug designations with descriptive and comparative statistics.

Results

Between 1983 and 2019, 124 orphan drug designations were granted by the FDA for compounds intended to treat 28 lysosomal storage diseases. Orphan drug designations focused on Gaucher disease (N = 16), Pompe disease (N = 16), Fabry disease (N = 10), MPS II (N = 10), MPS I (N = 9), and MPS IIIA (N = 9), and included enzyme replacement therapies, gene therapies, and small molecules, and others. Twenty-three orphan drugs were approved for th

SUBMITTER: Garbade SF 

PROVIDER: S-EPMC7141691 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature