Orphans Account For More Than A Third Of EU New Drug Approvals In 2022

Last year, the European Commission approved a record 55 therapies containing a new active substance, including 21 medicines for rare diseases. Among them were a number of gene therapies for indications such as cancer and hemophilia.

DNA Fault
Gene therapies were among the many rare disease products approved in the EU last year • Source: Shutterstock

The number of medicines and vaccines containing a new active substance (NAS) approved in the EU hit a new high last year, with 55 products receiving a centralized marketing authorization, compared with 52 in 2021, which itself was a record number.

Orphan drug approvals also set a new benchmark in 2022, with 21 medicines for rare diseases accounting for more than...

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