The one-shot gene therapy for hemophilia, Hemgenix, has been designated as an orphan drug in Korea.

On the 6th, the Ministry of Food and Drug Safety announced so through an orphan drug designation notice.

Hemgenix (etranacogene dezaparvovec-drlb), which is the first and only one-time gene therapy approved for the treatment of adults living with hemophilia B, was first approved by the FDA in November last year.

The drug can be used to treat adults with hemophilia B who: currently use Factor IX prophylaxis therapy, have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.

Hemgenix’s efficacy was confirmed t