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Article History

Accepted: 29 June 2026

First Online: 21 July 2026

Declarations

Conflict of interest: The authors report no potential conflicts of interest or financial disclosures that are pertinent to this article.

Availability of data and materials (data transparency): All data analyzed in this study were obtained from publicly available sources, including EMA databases, European Public Assessment Reports (EPARs), Orphan Maintenance Assessment Reports (OMARs), published literature, and commercial databases (data cut-off: 30 May 2026). All references are cited in the manuscript. Additional processed datasets are available from the corresponding author upon reasonable request.

Ethics approval: Not applicable.

Consent to participate: Not applicable.

Consent for publication: Not applicable.

Code availability (software application or custom code): Not applicable.

Author contributions: The first draft of the manuscript was written by E.G. and A.H.-S.; E.G. performed literature and data extraction; E.G. and A.H.-S. conducted data analysis and interpretation; and E.G. and A.H.-S. conceived and designed the manuscript.