The FDA has officially approved Takeda’s Orzeyful

The FDA has officially approved Takeda’s Orzeyful, the world’s first drug that boosts the orexin-2 protein to treat Type 1 narcolepsy.

Unlike older medicines that only relieve symptoms, Orzeyful targets the underlying biology of the disease — sudden daytime sleep attacks and the abrupt muscle weakness known as cataplexy. Two large clinical trials showed it was significantly better than placebo at keeping patients awake and reducing muscle weakness. The drug was generally well-tolerated.

FDA officials called it “the first medicine that impacts the underlying biology of the disease.” Patient advocates described the approval as a historic moment that expands treatment options.

Takeda has not yet announced the price, but analysts estimate it could range from about $142,000 to over $250,000 per patient per year. At the lower end, peak annual sales could reach roughly $2 billion.

Because the drug affects brain chemistry and reward-related networks, it must still undergo DEA scheduling review, which can take up to 90 days before commercial launch.

This approval opens a potentially multi-billion-dollar market for orexin-based sleep drugs. Major companies including Eli Lilly (which paid $6.3 billion for Centessa), Alkermes and Eisai are already racing to develop their own candidates. Alkermes is aiming for broader approval covering Type 1, Type 2 narcolepsy and idiopathic hypersomnia.