Fig 1.
A: Number of orphan drug designations (open bars) and FDA approvals (full bars) for compounds intended to treat lysosomal storage diseases by year. * indicates close of database: 10 May 2019. B: Overall number of orphan drug designations (open bars) and FDA approvals (full bars) by year. * indicates close of database: 10 May 2019.
Fig 2.
A: Orphan drug designations granted by the FDA for compounds intended to treat lysosomal storage disorders by year and specific disease. B: Orphan drug designations granted by the FDA for compounds intended to treat lysosomal storage disorders by year and pharmacological technology platform.
Table 1.
Withdrawn orphan drug designations.
Reasons for and time of withdrawal were not specified in the FDA database.
Fig 3.
FDA approved compounds for the treatment of lysosomal storage disorders (depicted as compound #disease), development times and market exclusivity.
A: Grey bars indicate drug development times, i.e. time from orphan drug designation to orphan drug approval by the FDA. Black bars indicate, if applicable, market exclusivity periods. (1)–systemic administration, immediate release (IR). (2)—ophthalmic solution (OS). (3)–systemic administration, delayed release (DR), adults. (4)–systemic administration, delayed release (DR), age 2 to 6 years. (5)—systemic administration, delayed release (DR), age 1 to less than 2 years. (6)—bioreactor 160 L. (7)—bioreactor 4000 L, 8 years and older.(8)—bioreactor 4000 L, all ages. B: FDA approved therapies for the treatment of lysosomal storage disorders by year of approval and pharmacological technology platform.
Table 2.
Mechanism of action of FDA approved small molecules (*) and small molecules in development, intended to treat a lysosomal storage disorder.