Data-Driven Research Needed to Ensure Timely Access to New Narcolepsy Treatments

Dr Helen Strongman shares her perspective on access to new narcolepsy treatments – as a person with narcolepsy, trustee and director at Narcolepsy UK, and health data scientist¹.

As a person with narcolepsy, I fight an everyday tug-of-war with my brain’s desire to yo-yo between sleep and wakefulness and my determination to lead a fulfilling life. Since my diagnosis, I have followed developments in our understanding of the biological cause of narcolepsy with interest and excitement. Thanks to Nobel Prize-winning Professors Mignot and Yanagisawa², I now understand that at some point in my teenage years my brain stopped making orexin, a molecule that helps to regulate the sleep-wake cycle. With further public and industry-funded research, experimental drugs are being developed that take over orexin’s role^3,4,5.  These drugs are called orexin agonists.

 In July 2025, Takeda announced that following completion of phase 3 clinical trials for overporexton (TAK-861), they plan to submit a new drug application with global regulatory authorities this year (2025)³. If approved, overporexton will be the first orexin agonist to be licensed in the US and countries covered by the relevant global authorities – this offers hope to people with narcolepsy worldwide.

In the UK, we might wait a long time before we have access to these new possibly life-changing treatments. My research has shown that uptake of new narcolepsy drugs is slow and varies greatly depending on the NHS hospital and area of the country in which you are treated⁶. This is partly due to a lack of data-driven evidence to guide treatment decisions and inform national guidance.^6,7,8,9

Everyone has the right to high quality evidence-based care and treatments – this is one of six rights recognised by Narcolepsy UK’s evidence-based Charter¹⁰. We need action!

¹ My work is funded by the National Institute for Health and Care Research (NIHR) through an Advanced Fellowship (NIHR301730). The views expressed in this blog are mine and not necessarily those of the NIHR, NHS, UK Department of Health and Social Care, my university (LSHTM), or Narcolepsy UK.

²Scientists who discovered cause of narcolepsy win Breakthrough Prize | New Scientist

³ Takeda Announces Positive Results from Two Pivotal Phase 3 Studies of Oveporexton (TAK-861) in Narcolepsy Type 1 | Takeda Pharmaceuticals

⁴ Alkermes Announces Initiation of Vibrance-3 Phase 2 Study Evaluating ALKS 2680 for the Treatment of Idiopathic Hypersomnia | Alkermes plc

⁵ Orexin Agonist (ORX750) – Centessa Pharmaceuticals

⁶ Trends and variation in issuance of high‐cost narcolepsy drugs by NHS England organisations and regions from 2019 to 2022 – van Someren – 2025 – Journal of Sleep Research – Wiley Online Library

⁷  Validation of algorithms identifying diagnosed obstructive sleep apnoea and narcolepsy in coded primary care and linked hospital activity data in England – ScienceDirect

⁸ Incidence and prevalence of obstructive sleep apnoea and narcolepsy in the UK: a population-based descriptive study | medRxiv

⁹  Uniting the UK’s Health Data: A Huge Opportunity for Society

¹⁰ Narcolepsy Charter – Narcolepsy UK