A rare disease treatment 'Joenja' has received orphan drug designation in South Korea.

The Ministry of Food and Drug Safety (MFDS) states this through the report on the designation of Orphan Drugs. Joenja (leniolisib), developed by the Dutch company Pharming group, is the first treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). The drug was approved by the U.S. Food and Drug Administration (FDA) in March of last year.

APDS is caused by a mutation in either the PIK3CD or PIK3R1 gene, essential to immune cell development and function. It occurs in 1 per 1-2 million.

APDS patients commonly develop autoimmunity and inflammatory symptoms. They may