FDA’s authorisation addresses the condition’s full symptom burden instead of just symptomatic relief.
The US Food and Drug Administration (FDA) has approved Takeda’s Orzeyful (oveporexton) as the first medicine to directly target the loss of orexin signalling that causes Narcolepsy Type 1. It also addresses the full range of symptoms that define the rare condition.
Dr Emmanuel Mignot principal US investigator in the medicine’s Phase III programme, said: “Until now, people have managed narcolepsy type 1 with treatments that target symptom relief.”
Instead of masking symptoms with stimulants or sedatives, the oral orexin receptor 2 (OX2R) agonist directly activates the same brain receptor that the body’s own orexin would normally stimulate, restoring the missing signal.
Currently, an estimated 120,000 people in the US live with this form of narcolepsy.
The approval is based on a clinical programme that includes the Phase III studies FirstLight and RadiantLight. Findings (1, 2) showed oveporexton significantly reduced daytime sleepiness and cataplexy episodes and greatly enabled improvements across the full range of symptoms of the neuropsychiatric sleep disorder.
[Orzeyful] marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment”
Julie Kim, President and Chief Executive Officer of Takeda
Julie Kim, President and Chief Executive Officer of Takeda, added that approval of Orzeyful “marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment.”
Eli Lilly is another big pharma player who is capitalising on the potential of orexin agonists. In April, the firm agreed to acquire biotech Centessa Pharmaceuticals for approximately $6.3 billion. The candidate is being developed for multiple sleep-wake disorders: narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia.
This follows the company’s executive appointments in neuroscience last November. Dr Carole Ho agreed to serve as Executive Vice President and President, Lilly Neuroscience. Dr Daniel Skovronsky is Lilly Research Laboratories’ new Chief Scientific and Product Officer, and President.
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