RNAi therapeutic 'Givlaari' has been designated as an orphan drug following its designation as the GIFT.

The Ministry of Food and Drug Safety (MFDS) recently announced this through the posting of the Orphan Drug Designation (ODD).

Givlaari (givosiran) had previously been designated as the 'Global Innovative products on Fast Track (GIFT)' by the MFDS.

This drug won U.S. FDA approval in 2019 and also received the accelerated approval from Europe's EMA.

Givlaari is a subcutaneous injection that targets the enzyme aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.