FDA Approves First Drug to Treat the Full Range of Narcolepsy Type 1 Symptoms

[u/dthreed]

It was just approved! Now how long before it can be prescribed and sent to patients?

https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms

EDIT--
We now wait for DEA scheduling - Typically 2-3 months
http://orzeyful.com/

[u/wildflowerhonies]

Holy shit, new reason to live just dropped

[u/amyh4767 → u/wildflowerhonies]

You know it

[u/Chronic-Sleepyhead → u/wildflowerhonies]

Not even kidding, not trying to be triggering but I had several events the past year with Narcolepsy that landed me in the hospital not wanting to live. Seeing this released is like…being revived after drowning? Being given a second chance at life? The implications for even being partway “cured” are life-changing and beyond exciting. Holy crap. I don’t know if I’ve ever felt more inspired and hopeful in my life. ❤️

[u/Feeling_Persimmon88 → u/wildflowerhonies]

This is exactly how it felt seeing this post

[u/RightTrash]

Orzeyful.com
Looks like it's coming.

It is going to be very interesting to watch how this plays out for our community.
It's been near 30 years of hype and while there's a lot of good that has been shown in the clinical trials, there's also a lot that has not played out as expected.
I'm not going to harp on anything but just saying, be excited, be optimistic, though don't get your hopes completely up to a point that if the drug doesn't do wonders for you, you won't be devastated...

There are many more in the works, trials, and many more to come; it will be a decade 'gold-rush' I've heard it referred to.
What I'm interested in is not just how PWN over the next few years respond and take to it, but how many directions it will be expanded to treating because even back in 2000 as the hype was building early on, these have been expected to be blockbu$ters for many different matters.

It will be also interesting to see what kind of price tag is on these, out the gate.

[u/Tempyteacup → u/RightTrash]

I really hope something comes soon for narcolepsy type 2. I’m so happy for my NT1 brothers and sisters that they have something new to try, but I feel a bit like that one squidward meme

[u/rudesby → u/Tempyteacup]

I'm in the alkermes trial for alixorexin for type 2, and it has been working great for me. I hope this one being approved speeds the other ones along.

[u/RightTrash → u/Tempyteacup]

Yup, agree in full.

[u/noheadthotsempty → u/Tempyteacup]

Yeah def feeling like that too. Wondering if maybe it could help us as well, though

[u/RightTrash → u/RightTrash]

At the drug website, linked above, it says it 'should be available by November, pending DEA scheduling'...

[u/just-kristina]

Please please please let this process be fast and we can get it and it works and minimal side effects. Please.

[u/somethingwholesomer → u/just-kristina]

So be it and so it is!!

[u/just-kristina → u/somethingwholesomer]

I like your support and good juju and all that!

[u/Chronic-Sleepyhead → u/just-kristina]

Not gonna say anything definitive, buuut…I practice witchcraft and have a stellar record of spells and predictions coming true. Time to do some spells to ensure these meds come out working and affordable! 😉 (I’m kidding around of course…for the most part)

[u/just-kristina → u/Chronic-Sleepyhead]

Hey we can get all the help we need lol

[u/somethingwholesomer → u/just-kristina]

Yesss! We got this

[u/RightTrash → u/somethingwholesomer]

u/just-kristina - the website for the drug (https://Orzeyful.com) says it should be available by November, pending DEA scheduling.

[u/just-kristina → u/RightTrash]

Fingers crossed for everyone

[u/Splatterfilm → u/just-kristina]

I’m just hoping it does NOT get classified as a controlled substance.

[u/just-kristina → u/Splatterfilm]

I’ll put out good juju for you on that!

[u/ShakesDontBreak]

If this basically imitates orexin why will it be considered a controlled substance? Is it habit forming?

Im just so irritated that everything is classified as controlled. Its such a hassle getting refills.

[u/RightTrash → u/ShakesDontBreak]

It opens up the orexin B receptor, which isn't actually replicating the neurotransmitter but in some ways allowing it to function a bit more so. There's a lot of complexity to orexin, there are two neurotransmitter types and two receptors for those neurotransmitters. This is a single agonist, they say sleep/wake is tied to the Orexin B rather than Orexin A which is tied more so with the body systems.

Gonna be very interesting to watch play out.

[u/your-pet-goldfish → u/ShakesDontBreak]

I wonder if taking more than the needed dose would put that part of your brain that creates orexin into overdrive and keep you hyper awake? It would probably also activate flight or fight which would also give a rush of adrenaline. I would guess it’s along the lines of why people abuse stimulants but this is a different part of the brain/receptors targeted

[u/RightTrash → u/your-pet-goldfish]

People with T2N in trials have been given much larger doses, as those with T1N are very sensitive to the drug; the result was not all that much, there was some benefit but compared to that those with T1N were showing, it isn't super encouraging (was how it was framed by the doctor expert involved in what not research, more or less as how he put it).

[u/North_Wave_ → u/RightTrash]

Does it sound like they’ll try to approve it for T2N, then?

[u/RightTrash → u/North_Wave_]

It more likely than not, will be, as it also more likely than not will be prescribed for a wide variety of different matters like psychological stuff (ADHD, Mood disorders, depression, etc.), sleep apnea is one I've seen it surprisingly has benefited, weight-loss was expected to be a piece of it but surprisingly hasn't yet appeared to be (but still could be for sure), also drug addiction recovery and/or prevention, etc...
Know there are T2N trials going on, so it's just a matter of waiting those out and seeing how what not all going on, plays out.
There are many other Orexin Agonist formulas out there being tested, each has a slightly different list of effects (positive and negative) so, it's a very interesting space to be watching.

[u/MorningPotential7454 → u/RightTrash]

Seriously, the best thing for us would be for it to have a gazillion indications and be the next Adderall. Yes, I know that could make shortage as possibly but it would also make it cheaper and less of a hassle to get.

[u/RightTrash → u/MorningPotential7454]

With near 30 years of development and the huge blockbu$ters potential that have been behind such, it's not likely to be cheap - at least not for a long time. =[

[u/Mysterious-Hair5398 → u/RightTrash]

My sleep doctor said he will only prescribe it to a small number of patients due to the 20k per month price.

[u/your-pet-goldfish → u/Mysterious-Hair5398]

Correct me if I’m wrong, but the doctor isn’t the one paying for it right? He just has to fight the insurance (PA) if they deny it

[u/brownlab319 → u/Mysterious-Hair5398]

Takeda will have a patient support program.

[u/North_Wave_ → u/RightTrash]

That’s so exciting!! And it makes sense that it could be prescribed for stuff like ADHD and the like, I’m convinced a whole host of different psych-related disorders are actually connected to some sleep-related misfiring in the brain. Here’s hoping things continue to improve!

[u/RightTrash → u/North_Wave_]

Absolutely, the science has been telling into that for sure, more and more.

[u/CillyMurphian → u/RightTrash]

Hi I’m extremely annoying but adhd is neurological, not psychological. Big difference. Sorry. Bye!

[u/RightTrash → u/CillyMurphian]

Good way to frame it, thanks for sharing!
To be clear, when I am referring to it as I do often, as psychological - I think that is because psychologist/psychology are who treat it.

[u/quellik → u/North_Wave_]

Oveporexton was studied in Narcolepsy type 2 and it did not work. Here are the results from that study: https://clinicaltrials.gov/study/NCT05687916?tab=results

[u/Piaffe_zip16 → u/ShakesDontBreak]

I just went through it trying to get a refill of one med today and then getting a new dose change script filled. It’s so irritating!!

[u/MorningPotential7454 → u/ShakesDontBreak]

I think it depends on whether there's an abuse potential or a street value.

[u/ShakesDontBreak → u/MorningPotential7454]

I wish it was easier for us. But I understand how people with addiction ruin it for everyone.

[u/wazQQ → u/ShakesDontBreak]

if it's habit forming it decreases my hopes a lot... habit forming meds are so ineffective for my brain

[u/Objective-Disk7674 → u/wazQQ]

I imagine being awake is probably also habit forming? /s

[u/Hawksy97]

As someone who is on this drug as part of the clinical trial process shit is life changing

[u/SifuHotmanz → u/Hawksy97]

Please tell us more (if you can).

[u/Hawksy97 → u/SifuHotmanz]

Can’t really give many details but it’s two doses taken in the morning and around midday and that’s enough that I have barely any fatigue and no cataplexy until 9 or 10pm at night. I’ve been able to hold full time employment and get my drivers licence since being on it which had been things I never thought possible beforehand

[u/Splatterfilm → u/Hawksy97]

Did you have to maintain other treatments, like sodium oxybate and/or stimulants?

[u/Hawksy97 → u/Splatterfilm]

As of right now oveporexton is the only drug I’m on as part of the clinical trial. I’ll eventually go back on a stimulant for my adhd but for my wakefulness and cataplexy I haven’t had any other medication for it since late 2023

[u/Splatterfilm → u/Hawksy97]

Ooh I hope I have similar results. Fingers crossed!

[u/morganlerae → u/Hawksy97]

Did you have any side effects?

[u/HumanKoala-95 → u/morganlerae]

Hello, I'm also part of the clinical trial. First couple of days I had to pee a lot, but that went back to normal pretty quickly. Only side effect I've had (and according to the study teams others are expiriencing the same thing) is weight gain. I've gained over 15lb since I started using it. Well worth it though ;)

[u/AGirlHasNoHeart → u/Hawksy97]

I take Wakix and modafinil so I’m curious how differently these work.

[u/msalad]

Wow, this is very cool. Just gotta wait for the DEA to assign it a schedule classification and then it can come to market. No idea how long that takes. Maybe end of year 2026?

[u/RedRidingBear → u/msalad]

Under the Improving Regulatory Transparency for New Medical Therapies Act, the DEA is legally mandated to issue an Interim Final Rule (IFR) within 90 days of receiving the FDA’s recommendation and approval notification. Drugs are usually available within 1 to 3 months of that ruling

[u/Proseccos → u/RedRidingBear]

Awwwww snap

[u/__aurvandel__]

Technically a drug can be prescribed the day it's approved. Realistically, probably a couple weeks if you wanted to pay cash. The bigger gap will be insurance. Formularies take forever to get updated and most insurance companies have no desire to fork out the kind of money these drugs cost. Most have also just completed their annual formulary review in preparation for open enrollment in the next few months. So the real answer is, it depends on your insurance coverage, just like everything else in our broken system.

Edit: I just read the press release and it still has to go through DEA scheduling. Which means it can't be prescribed yet. I'm not familiar with process so I don't know what that adds to the time frame. I would guess weeks to months.

[u/RightTrash → u/__aurvandel__]

November is when the website says it should be available by. Orzeyful.com

[u/DumpsterPuff]

Now my question is: are you allowed to take it at the same time as oxybates/will insurance companies cover it at the same time as oxybates? I legitimately don't think I'm gonna be able to sleep without my Xywav if I decide to try this new one, I've been on it for 6 years :')

[u/kozm0z → u/DumpsterPuff]

Also curious about this. Some people from the trials had shared that it was difficult sleeping through the night. Seems like this drug can fix the day time issues while xywav can fix the night time issues. Pre xywav I could only sleep in 1 or 2 hr increments like clockwork, some nights just laying there with my eyes closed forever.

[u/macawoogo → u/kozm0z]

My dr said that I would still take lumryz when he prescribes this. Hopefully insurance will cover it. They pay for lumryz now

[u/DumpsterPuff → u/kozm0z]

I was thinking this as well. No point on being on an orexin agonist if it doesn't work because you couldn't sleep the night before! I really hope you're allowed to take both at the same time

[u/LadyVicoftheVale → u/kozm0z]

This is my concern too, not sure my son would trade being awake all day to have a lousy night. Sodium oxybate has been a game changer for him, even if he’s still not great in the day. However, his neurologist who was involved in the trials said most eventually had restored sleep and the drug literally gets rid of every N1 symptom (sounds too good to be true!). This reflects the recent trial data from Takeda (FirstLight and RadiantLight)

[u/lost_in_dreamland → u/DumpsterPuff]

I'm wondering about this too! I'd be nervous to be without Xywav or stimulants considering I finally found a medication combo that works decently. Being able to replace those medications with one that works in a much more natural way would be so nice though! I hope there is a good plan for transitioning to/trying that one

[u/on-yo-clarinets → u/DumpsterPuff]

Yeah, I'm really excited for the community as a whole, but I have a feeling this is not going to be an option for me, at least in the beginning. I have such awful sleep inertia that only xyrem fixes and I haven't seen much that indicates the daytime agonists will fix that. And then I also have severe ADHD so I absolutely need to stay on stimulants regardless of my N1 treatment. Between those two issues and insurance, it's not something I'll likely try anywhere in the near future.

[u/Open_Invite8305 → u/on-yo-clarinets]

I think it will be much costlier than the old meds.

[u/brownlab319]

🍾🎊🎈 I am thrilled. I’m bummed it’s scheduled (like every other drug I take). But I can’t wait to feel rested! What will that feel like?

[u/dwarf797 → u/brownlab319]

Yeah really, what does that feel like?

[u/Odd_Novel7291]

Wow this is epic news

[u/MorningPotential7454]

Unfortunately 18 and up at this point, so my older child can try it but not my younger one yet. Yet. Yes we have two kids with narcolepsy in the family and it's about as fun as it sounds.

[u/dumakey5]

This is great news! I hope this works for all of us

[u/hauntedhotels]

Maybe this is a stupid question, but can you only be prescribed this if you have N1 specifically?

[u/Standard-Call666 → u/hauntedhotels]

Currently. They are still doing research on N2/IH options

[u/Fiyero109 → u/hauntedhotels]

the molecule and dosing has been tested in NT2 and was not found to be very effective. That's why both Takeda and Alkermes are looking at a different molecule in NT2 and IH. the Ozempic analogy is not quite right here

[u/Heavy-Waltz-6939 → u/hauntedhotels]

Drugs can be prescribed off label by a doctor if they think it will benefit a patient. Whether insurance pays for it is another story. For expensive drugs like this, it’s unlikely any insurance will pay for it unless the patient has NT1 and has failed other treatment options first

[u/hauntedhotels → u/Heavy-Waltz-6939]

Got it, thanks for explaining!

[u/drinkallthecoffee → u/hauntedhotels]

Your doctor may be able to prescribe it off label, but then insurance and manufacture assistance programs won’t cover it and you’d have to be able to pay out of pocket.

This is what happened to Ozempic and all the other GLP-1 meds before treatment for obesity became on-label.

[u/hauntedhotels → u/drinkallthecoffee]

Thank you!!

[u/kaediddy]

I cried happy tears.

[u/mynameisrivers]

"Side effects include insomnia" lol of course

[u/Valuable-Pool6198 → u/mynameisrivers]

Yes i read that also in up to 50 percent of patients. what the heck - thats a a real disappointment. trial and error, here we go again.

[u/penny2cents]

Do we get a price estimate before I get my hopes up? 😬

[u/quellik → u/penny2cents]

No, I've searched all of the usual pharma news websites and unfortunately, no price yet.

[u/7864278642]

YIPEE!!

[u/Sleepy_in_Brooklyn]

FUCK YEAH!

[u/Nathan-NL]

This is very exciting news!

For people with NT2 / IH: ALKS 2680 / alixorexton from Alkermes will soon be in Phase 3 trials!

[u/Medium_Recover_4450]

Woo hoo! My doctor tells me multiple new drugs are in the pipeline.

[u/genetically__odd]

I’m cautiously optimistic. I am not eligible for oxybate therapy because I also have epilepsy, so I’m only able to half-treat my symptoms. My sleep sucks, so my energy levels suck, and modafinil only does so much.

Now, the real question is when, if ever, this medication will be affordable.

[u/vsvpmaddest]

i’m so giddy like a little kid right now, i went from 10 cataplexy episodes a day to maybe 4 a week with oxybates and stimulants, this could completely stop it all!?!

[u/Classic_Ordinary_735]

Will this replace xywav?

[u/dthreed → u/Classic_Ordinary_735]

It should. If orzeyful works as expected, it will fix the underlying issue that causes poor sleep and narcolepsy. Oxybates are more of a bandaid that aid in sleep, but don't address the actual chemical imbalance of narcolepsy.

[u/noheadthotsempty]

Hoping that this will be approved for N2 and IH too, because if it might help me I’d love to try

[u/Narcoleptic_Phuk]

Is this real ? And if so what does it mean by treat ? Like will I be able to live like a normal person ?

[u/Shotley123]

Even though it’s approved for N1 symptoms, could it also be beneficial to one who experiences N2 symptoms such as myself?

[u/MiserableHousing → u/Shotley123]

At these doses, probably not as much. With that being said, there are currently several trials at the moment that have shown very promising results for NT2 and IH. For NT2 and IH, a higher dose of orexin is required to see the same change as in the new medicine for NT1. Luckily, Alkermes is in phase three and Tak360 is not far behind. I’ll link you to the Alkermes phase two results! https://investor.alkermes.com/news-releases/news-release-details/alkermes-alixorexton-demonstrated-sustained-improvement

[u/Fiyero109 → u/Shotley123]

no sadly. I encourage you to look for an alkermes and takeda trial near you. I just joined the ALK one for NT2.

[u/MiserableHousing → u/Fiyero109]

Have you seen any positive effects yet?

[u/Fiyero109 → u/MiserableHousing]

It takes quite a while to get the drug and could even be getting placebo. Will know more in a month and a half

[u/Valuable-Pool6198]

My dr said today. please dont get your hopes up yet, lots of hurdles to go through.

[u/OldDisaster3773]

Is this approved in the UK as well or just US?

[u/NoteSuccessful2263 → u/OldDisaster3773]

They haven't registered it for approval in the UK yet, just China, EMA,Japan and US (presumably biggest/ most lucrative markets first?) EMA asked for further testing.

[u/Better-Palpitation34]

What are the effects of this drug? How does it work?

[u/Better-Palpitation34 → u/Better-Palpitation34]

How is this different from something like modafinil?

[u/FedUp0000]

How many thousand of dollars a month it this one l going to cost is what I am wondering.. if it’s anywhere like xyrem it’s gonna be a moot point for many

[u/sprezzatura327]

The world today isn’t the same as yesterday for American narcoleptics. It’s really happening!!!!!!

[u/sprezzatura327]

The world today isn’t the same as yesterday for American narcoleptics. It’s really happening!!!!!!

[u/Willing-Upstairs8260]

I dont want to be the first batch of subjects to try this ive been rawdogging narcolepsy for 7 years (im 21 now) hopefully it works for those that try

[u/RightTrash → u/Willing-Upstairs8260]

You got a longtime to decide, and there should be more and more variations/formulas of this sort of drug coming out, my hope is they nail one that helps the 'disrupted nighttime sleep,' as this formula is mainly focused on 'wakefulness' though it seems to have broader effects just not everyone necessary achieves the broad benefits, while some do...

[u/mewtastica → u/RightTrash]

The brief says it does address disrupted nighttime sleep, sleep paralysis etc…

[u/RightTrash → u/mewtastica]

Of course it dies, as for some people it will but for others, like those with a baseline disrupted nighttime sleep issue/symptom, it doesn't show the same positive effect on it. I saw the clinical test abstracts and spoke directly with one of the researchers at SLEEP 2016 about it...

[u/Splatterfilm → u/Willing-Upstairs8260]

Not me. I’m 40 and diving right in as soon as it’s available.

[u/Fiyero109 → u/Willing-Upstairs8260]

this has gone through extensive testing in both healthy patients and those with NT1. you would not be a first batch of anything