US FDA approves nasal spray alternative to EpiPen for allergic reactions
reuters.com
reuters.com
> The shelf life of neffy is 30 months and allows for temperature exposure up to 122°F (50°C), making it a potentially effective treatment if left in a car or outside for a length of time. If accidentally frozen, neffy can be thawed and administered. [1]
The need to keep Epi-Pens below 77 degrees F (86F for short periods) is extremely constraining for something that you are supposed to carry with you at all times. Even keeping it in a jeans pocket next to your skin may not be acceptable, technically. If it's hot outside, you can't leave it in your car or even in a bag you're carrying. You're supposed to carry a thermos bottle with you the whole day every day or something? Obviously very few people do that. This is going to be far better for that reason alone. And double the shelf life is very welcome too.
[1] https://ir.ars-pharma.com/news-releases/news-release-details...
> Forty EpiPen and EpiPen Jr auto-injectors that had been expired for 1 to 50 months were tested. All 40 auto-injectors contained more than 80 percent of the labeled dose
What that third-party test shows is that if all you have available is a supposedly 4-year past-expiry pen and it's been kept at all the wrong temperatures, you should probably still use it in an emergency because there's a good chance it will still work. Not a big enough chance that they recommend using expired pens, but enough that if the choice is probably dying or using it, you absolutely should use it.
Edit: Found a picture of an official case. Carries two doses and looks roughly similar in size to two Auvi-Q doses: https://andersresearch.substack.com/p/aquestive-therapeutics...
I bet a third party could make a smaller case than that, though. I also expect that neffy is much lighter, as Auvi-Q has a battery and a speaker and a needle and a spring and whatnot.
Going to hospital afterwards (to be monitored for late phase reaction and receive high dose antihistamine and hydrocortisone) usually seems to involve putting a cannula in my hand for IV medication which is pretty well guaranteed to be much more unpleasant.
The hard part being, front the patient POV we didn't know if it's in excess of precaution or something really critical.
There's a decent selection of thermo cases that are small enough to make it manageable, but it's definitely a complicating factor, especially for smaller kids.
In my country, injecting a drug via needle is a medical activity only the patient himself, doctor and the nurse instructed by doctor can perform by law.
For EpiPen, the regulation is slightly relaxed so the paramedics, parents/childcare workers/school teachers responsible for the child patient can also use it.
But when the EpiPen is needed, the patients often can't administer himself.
This will allow us to be much more relaxed, since the risk of anyone panicking when administering is lower, the risk of doing it wrong is significantly lower, and the stigmata is also lower compared to a needle.
I've been eagerly awaiting this.
This is considerably lower than the non-insured cost of an Epi-Pen or generic equivalent[0]. Hopefully this spurs some competition and makes the whole market more affordable. I needed to use an Epi-pen in college, and though I never took issue with needles I'm glad than an alternative is available for those fearful of them.
(I didn't feel it. I was about to pass out. But I remember looking up from the floor while administering and seeing my RA terrified. He was so scared of needles that if I had lost consciousness, he wouldn't have administered for me!)
[0]https://www.talktomira.com/post/how-much-does-an-epipen-cost
[0]: https://ir.ars-pharma.com/news-releases/news-release-details...
I wouldn't be surprised, though, if insurers continue to push generic epinephine injectors ($15-$30) merely from a cost perspective.
https://www.cbc.ca/news/health/epipen-shortage-canada-1.3296...
The actual price of 2pk epipen is $109.99 and it lasts many years. As I mentioned in another comment, "Forty EpiPen and EpiPen Jr auto-injectors that had been expired for 1 to 50 months were tested. All 40 auto-injectors contained more than 80 percent of the labeled dose" https://www.foodallergy.org/fare-blog/expired-epinephrine-ca...
Unless some institution forces you to pay more (Eg schools and allergy shot providers checking expiry dates) the actual cost is ~$25/year if you don't have decent insurance and ~$0 if you do
Paying hundreds of millions of dollars to settle antitrust claims sounds like a monopoly to me.
https://www.biopharmadive.com/news/how-mylan-ended-up-with-a...
https://kffhealthnews.org/news/behind-the-epipen-monopoly-lo...
https://www.courthousenews.com/10th-circuit-asked-to-revive-...
https://www.reuters.com/legal/litigation/pfizer-pay-50-mln-s...
https://www.nbcnews.com/business/business-news/epipen-maker-...
Nope, first "there is no monopoly". I don't give a shit about 2016
https://www.cvs.com/content/epipen-alternative
(Sometimes you could need two doses in the event of an emergency, according to doctors.)
I know this because I've been buying them for years without use (thankfully), on the off chance my son has a life threatening reaction to eggs.
The guy had some kind of natural aversion combined with an apparently traumatic memory from childhood of being held down and poked multiple times (probably due to their resisting) by nurses.
We shouldn’t be dismissive about how sub-optimal it is that children are routinely held down and stabbed, against their protests, by authority figures. There’s nothing surprising about people growing up from this and not trusting medical experts.
Irrelevant to the topic, COVID vaccines were developed with new technology that uses synthetic materials.
I am over simplifying a lot here, but the point is that sometimes you need to get that dead matter into the bloodstream to start the auto-immune processes.
To your point, it does depend: there are needle-free options available for some vaccinations currently, though in many cases children are nonetheless forced to take the version that they beg to decline.
The children have the least say in where the research funding goes, negotiating reimbursement contracts between payors and providers, planning pharma development pipelines… My opinion is that we’re failing them.
The reasoning I've heard is since Epinephrine constricts blood supply to the region and you can kill the tissue in an area with small blood vessels like your fingers.
Anyone aware of what the risks of a spray would be in similar contexts? I imagine stabbing a finger is not a risk here, but what about the spray getting anywhere other than the nose like in eyes etc....
EDIT: Looking at the product page https://ars-pharma.com/product/ it looks a lot like a Naloxone nasal spray so, I suppose its easier to position it in nose (not an inhaler like thing as I was imagining).
For epilepsy, rescue meds to prevent one seizure from triggering another have started coming in the form of nasal spray for at least 5 years already. Before that, outside of hospitals you either had to put a pill under someone’s tongue or cream in their butthole. You couldn’t put it under their tounge during a seizure out of risk they’d bite your finger (I’m currently recovering from a tongue bite I got after having a seizure while alone 8 days ago & it’s pretty miserable. Amazing the amount of power the teeth/jaw can have) and let’s just say the butthole option is unappealing.
My second question is should I invest in the company?
This seems like it has the potential to sell a ton. And if margins are anything like the nasal rescue med for epilepsy they are massive. The epilepsy nasal sprays are about $300 each without insurance, the same med in pill form is about 10 cents. (Yes, I’ve struggled a lot over the years with the level of privilege and inequality that having access to them reflects/perpetuates.) Their stock has tanked since going public for reasons I’ve yet to find time to research.
My bet is that FDA took 5 years longer than reasonable to clear it.
The initial submission for approval was in October 2022. The FDA replied in September 2023 asking for two specific additional pieces of information. The company submitted the new information in April 2024.
* https://ir.ars-pharma.com/news-releases/news-release-details...
* https://ir.ars-pharma.com/news-releases/news-release-details...
A non-snarky answer for "what took so long", is that nasal dosing is challenging, especially since too much drug can cause adverse responses as well.
ARS itself seems to have been founded in 2015. 7 years from company founding to submission (and 4 years to being granted fast track) is maybe... a little slow? But at the same time, how quickly do you expect a novel delivery mechanism for a life saving drug with an existing, effective and widespread alternative to go?
* https://www.sec.gov/Archives/edgar/data/1671858/000119312523...
I only know of their existence because I’ve used them for epilepsy personally. Are there any other contexts they’re used other than that and now this? They’ve obviously been used outside of emergencies for decades.
If it was completely revolutionary when it was brought to market for epilepsy then it makes sense.
The first drug approved using intravail was tosymra (for migraines) and was approved in 2018. Intravail itself seems to have been around since at least 2006.
This drug (neffy) also uses intravail.
I would speculate that intravail (or an excipient like it) is probably required for rapid/acute/emergency situations. My expectation is that the first approval of a nasal drug using intravail would have greatly reduced the hurdles for any subsequent drug.
I don’t know enough about their history.
> ARS Pharma will offer neffy at a price of $199 for two doses via digital pharmacy sites like BlinkRx and GoodRx for eligible patients whose insurance plans do not cover neffy. Some commercially insured patients can access the treatment at $25 for each filled prescription of two single-use neffy devices through a co-pay savings program.
But maybe not. Because folks with uncontrolled seizures need rescue meds more often than folks with life-threatening allergies need epi-pens.
Would be interesting to see the data on the potential demand for both products and how they compare.
Silverback had raised a pile of cash to push through a cancer drug, but saw their development programs stall out due to poor results.
https://www.fiercepharma.com/pharma/silverback-admits-defeat...
As for expected market size - you can look at their investor deck. Slide 29(of 33) for their projected unit sales (in probably the most awful graph I've ever seen). You can look at slide 28/33 for market share data of other newly released nasal drugs.
https://ir.ars-pharma.com/static-files/a2079aad-1856-42f9-90...
If the school could buy and store a reasonable number (funded, on a fractional basis, by the insurers of the students who need them) and use them as needed (with replacement paid for by the recipient’s insurance), the students’ lives would be simpler and a lot less money would be spent.
They all come home at the end of the year, expire, repeat.
So instead of the school managing and restocking a reasonable number, the parents restock one each year.
So:
- if school stocks them for 8 kids: they need to replace 16 every year
- if the 8 kids families supply the stocks: they need to, in aggregate, replace 16 every year
Same amount. Obviously better if the school pays, but I'm not understanding the "reasonable number" part.
A kid that regularly eats peanut butter but is very sensitive to bee stings doesn't need to plan to avoid strawberries.
And at some point, training the on site responders to inject from a vial is going to be pretty worthwhile.
Except if there’s overlap in the cause of the incidents that necessitate the usage of the device. Which is very likely in the case of a food allergy driving the event.
Imagine being the principal and trying to explain, with a lot of pretty charts and statistics, why you only hand 6 devices for 12 child patients.
- centralising all the devices in one point and have one or two trained professionals do the injections, which means a significant delay in case of emergency
- or training every professor to do injections, which isn't hard in theory, but time and cost consuming, puts more burden and restrictions on the staff (how do you deal with a 60yo math teacher who just wants to teach and not manage health emergencies ?)
And we're not going into the kids and the school staff properly communicating to get the person in charge to shoot the pen when the kid feel they need it.
Is that an option? If you're an adult supervising kids over long enough time you will have an emergency. I remember at least 3 decent ones from primary school.
I'd assume a teacher usually doesn't have the right to give medication to a kid, at least that's the rule we had when discussion with the school.
Obviously, yes.
> I'd assume a teacher usually doesn't have the right to give medication to a kid
We're talking here about emergencies and epipens. That's a bit beyond the "right to give medication" and in the good Samaritan laws territory.
This is the primary reason it's the kid and the parents who are in charge, that's the discussion we had with the school staff.
The only thing that I’m suggesting should change is to have a pile of epinephrine auto injectors (or nose sprays) supplied by the school, so the parents would instead just supply the instructions.
We were in one that couldn't provide meals (kid comes with it's own lunch, and they can't touch anything else) and individual medication was on a special cabinet in the classroom, but accessed by the kids.
TBH it felt more reassuring to us, as we only had to care about our kid properly reacting, and not about a whole chain of events we have no idea how well a generic school prepares for.
I'm still looking for the safety and efficacy study done. I also haven't yet found the justification for fast tracking this. An alternative to an epipen injection seems reasonable, but why rush it through when we already have the epipen?
[1] https://www.fda.gov/news-events/press-announcements/fda-appr...
I think they need some differentiating shape or like assigned danger colors for the most common emergency nasal applications.
> Of these, only two studies in rats were able to provide results that can be seen as an indication for direct transport from the nose to the CNS. No pharmacokinetic evidence could be found to support a claim that nasal administration of drugs in humans will result in an enhanced delivery to their target sites in the brain compared with intravenous administration of the same drug under similar dosage conditions