FDA approval ignites orexin drug race
Takeda's orexin-mimicking drug Orzeyful won FDA approval in August for narcolepsy type 1, with experts dubbing the class the 'GLPs of neuroscience' for its broad potential.
Takeda secured FDA approval in August for oveporexton, a drug that mimics a brain protein called orexin. Marketed as Orzeyful, it is the first approved medicine targeting the underlying biology of the uncommon sleep disorder narcolepsy type 1.
Experts are virtually unanimous that orexin mimics have much broader potential, with several referring to them as the 'GLPs of neuroscience.' These drugs could find use in other sleep disorders, fatigue, cognition, and mood - broad societal problems that present potentially massive markets.
The orexin story began in the late 1990s with the peptide's discovery by separate research groups. These hypothalamic peptides play a big role in alertness, acting as a master switch for arousal circuits.
Researchers quickly connected this system to narcolepsy. A key moment came in 2007 when stimulating the orexin system alone was found to be enough to wake sleeping mice. Takeda's own orexin work dates to the late 1990s, spurred by the understanding of the disease's cause. The development path included setbacks with earlier formulations, but the company ultimately succeeded with oveporexton, moving from first-in-human to approval in about five years.
The approval of Orzeyful follows Eli Lilly's $6.3 billion acquisition of sleep specialist Centessa Pharmaceuticals in March. Analyst Danielle Brill of Truist Securities said the bet and Takeda's approval are a big validation for the bull case on this class, which could have so much potential and could explode way beyond just narcolepsy.
Realizing these ambitions requires more clinical data. Experts suggest the drugs' greatest potential right now is for people with hypersomnia disorders, and that they could help those with neurologic disorders. Even confined to narcolepsy, the impact could be tremendous, as current medications reduce sleepiness but do not restore full function, while new data suggests these medications restore alertness much better.
Takeda is pushing forward to stay ahead of competition, having seen positive effects on mood, attention, and fatigue in trials and with an earlier-stage orexin agonist in its pipeline. Reflecting on the journey from a neglected condition, one researcher noted these drugs make a huge difference.
