|
BOSTON, April 29, 2026 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of oveporexton (Takeda Pharmaceutical Co., Ltd.) for narcolepsy type 1.
“Current treatments for narcolepsy do not address the underlying cause of the condition, and many patients report incomplete relief from narcolepsy symptoms despite multiple medications,” said ICER’s Senior Vice President of Research, Foluso Agboola, MBBS, MPH. “ICER’s analyses found that oveporexton, the first medication for narcolepsy type 1 that directly addresses the underlying cause of the condition, is effective in promoting wakefulness, improving quality of life, and appears to offer better health benefits than current options. Pricing choices that balance innovation rewards while addressing long-term value and patient access will be important topics at the public meeting.”
This Evidence Report will be reviewed at a virtual public meeting of the Midwest CEPAC on May 14, 2026. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
Register here to watch the live webcast of the virtual meeting.
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Clinical Findings
ICER found that oveporexton has at least a small but likely substantial net health benefit compared to no pharmacological treatment, resulting in an ICER rating of B+.
Compared with other medications such as modafinil/armodafinil plus venlafaxine, sodium oxybate, and pitolisant, oveporexton appeared to be more effective at promoting wakefulness. However, due to the limitations of currently available evidence, there is more uncertainty around the magnitude of improvements for other outcomes as well as around long-term safety. Therefore, when compared to these other medications, ICER gave oveporexton a rating of C++, indicating at least comparable but more likely a small to substantial net health benefit.
Key Cost-Effectiveness Findings
Oveporexton has not yet been approved by the FDA for narcolepsy type 1. The manufacturers have not yet announced a US price for the therapy if approved. ICER calculated a health benefit price benchmark (HBPB) between $50,400 and $59,400 per year.
ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.
| 
