Dr. Anne Marie Morse on the Significance of the FDA's Approval of LUMRYZ for Pediatric Patients With Narcolepsy

Last week, the U.S. Food and Drug Administration (FDA) approved Avadel’s LUMRYZ for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy. DocWire News spoke with Dr. Anne Marie Morse about the significance and benefits of this approval, and why the approval “is a true celebration” in the narcolepsy community.

Talk to us about the recent FDA approval of Avadel’s LUMRYZ for the treat of pediatric patients, 7 years and older, with narcolepsy. What is the significance of this approval?

Looking at the landscape of the diagnosis first of narcolepsy, this is a pediatric onset condition. We know this. We know that it most commonly is occurring between the ages of 5 and 16 years of age. However, it’s unfortunately burdened by the significant delay in diagnosis. You know what else it’s burdened by? The significant delay in appropriate treatment. We are depriving our children who have this condition from living their lives as fully as possible. When looking at the fact that we’re seeing interval growth of the number of medications now with an FDA indication for pediatrics, it is a true celebration. We are seeing the approval of Lumryz on the heels of just having another approval of another pediatric indication of Pitolisant for six years and older for excessive daytime sleepiness. You now have Lumryz with the approval, along with the twice-nightly oxybates of seven years and older for the treatment of excessive daytime sleepiness and cataplexy.

When we reflect on just the pentad of symptoms of narcolepsy, which don’t be fooled, this is not the extent of what people with narcolepsy go through. This is literally the tip of the iceberg. It is including excessive daytime sleepiness, cataplexy, sleep paralysis, sleep-related hallucinations, and disturbed nocturnal sleep. By being able to have the approval where a parent can oversee the dosing of the medication being done successfully, and it’s at the beginning of the night without any forced arousals, we are now not only honoring the fact that they innately are already having disturbed nocturnal sleep, but we also are not adding to the burden of having to have a forced awakening that many times is not just the patient, it’s the entire family. This is also a reflection of what the FDA so wisely bestowed upon Lumryz, which is an orphan designation of exclusivity for clinical superiority because it is not causing a forced awakening in individuals who have a chronic neurologic disorder.

Now, with all this stated, it is pivotably important for people to hear me say all three oxybates are tools in your tool about to treat pediatric and adult patients with narcolepsy because of the fact that we need to personalize care. So all three have a role for our patients. And the reason I state this is because after looking at the fact that we have oxybates as a treatment available since 2002, over 20 years, and reflecting on only 20% of individuals living with narcolepsy have either received, or are currently on, or even been offered. This now should provide even a greater degree of comfort to be able to introduce the conversation around oxybates because now I have the ability and the flexibility to treat more patients with the best treatment for them. It’s not saying oxybate for everyone, it’s saying you now can select the Oxybate that is best for the patient you’re partnering with.

And Lumryz is introducing a once nightly oxybate for children seven years and older, who now we don’t have a forced awakening. So I want to remind people, most patients requiring polypharmacy, it’s generally not one size fits all. It generally is introducing a host of different medications and making sure we are honoring what the patient actually is asking for, which is a better quality of life. Stop using [inaudible 00:04:00] to measure their disability and start allowing for them to not just survive, but thrive and make sure you’re considering which treatment plan is going to be most appropriate for your patient at this time, and making sure you’re reconsidering as they’re getting older and being able to do more things, continuing to respond with augmenting or personalizing that therapy to exactly what their goals are.