‘Amvuttra,’ the first new drug for amyloidosis with polyneuropathy to be introduced since ‘Vyndaqel,’ has been designated an orphan drug in Korea.

The Ministry of Food and Drug Safety announced so through a recent notice of orphan drug designation.

siRNA therapy Amvuttra (Vutrisiran) is administered subcutaneously at once every three-month intervals. It inhibits the production of wild-type and mutant-type transthyretin (TTR) by targeting and silencing specific messenger RNA.

Its efficacy was demonstrated through the Phase III HELIOS-A study that evaluated the efficacy and safety of Amvuttra in 164 patients with hATTR amyloidosis in 22 countries.

The pati