The FDA’s Neffy rejection: A story of balancing regulation and patient need
As a MedTech Pulse reader, you’re used to us bringing you stories of new health products approved for patient use. This story is a little different.
Today, we’re featuring the story of a new product, the epinephrine nasal spray Neffy, which was recently not approved by the FDA.
Why are we telling you a story about a product that failed to get approval?
Because this move from the regulatory agency came as a big surprise to Neffy’s developers, the patient community waiting on the product, and the broader healthcare industry. The decision came after the agency voted to recommend approval for Neffy back in May. For the FDA, a rejection following an approval recommendation is quite rare.
Join us to hear about Neffy, the common medical device it was supposed to supplement (or replace), and what we can learn from this story’s fallout.
Who needs an epinephrine nasal spray anyway?
If you have a child—or if you know someone with a life-threatening allergy—you’ve likely seen an epinephrine autoinjector like the EpiPen.
Neffy delivers that same drug for that same purpose. But it doesn’t require users to inject themselves or a loved one with a needle during a crisis.
ARS Pharmaceuticals and the allergy community responded with frustration.
“Our community believed this innovation would finally come to the more than 10 percent of Americans with life-threatening food allergies, but instead, the FDA will force us to wait even longer,” said Sung Poblete, CEO of food allergy advocacy group FARE.
What it comes down to is a tricky case of unmet patient needs. Though epinephrine auto-injectors are effective, life-saving devices, patients and families have been asking for needle-free options for years.
Especially when it comes to children, the fear of needles can keep them from administering life-saving medication. Hesitation in administering epinephrine can be the difference between life and death in anaphylaxis scenarios.
Plus, epinephrine autoinjector shortages have plagued allergy sufferers since 2017. Neffy presents a solution in the form of a simpler product design—one less likely to face the same shortages.
The uncertain path ahead for Neffy
For now, ARS hopes to resubmit Neffy to the FDA in early 2024, hoping for approval by the end of next year.
The FDA is requesting additional data from ARS, citing that the current trial data does not make them confident that the treatment would be effective.
Viatris, the EpiPen manufacturer, also filed a petition requesting more data from the ARS team.
Since epinephrine has been used in medicine since before the FDA, the pathway to approval for these products has been a little different than for devices that use new drugs.
ARS CEO Richard Lowenthal said the company was being asked to do a repeat-dose study, something the company was already prepared to do—but as a post-marketing requirement.
The new study will compare repeat doses of Neffy to repeat doses of injected epinephrine in patients with nasal allergy symptoms.
Our perspective: Innovating to upgrade already-effective treatments
Some of the most exciting innovations in the medical world involve completely new treatments (see our next two articles for that). But in discussing the Neffy situation, we hope to bring attention to an important other pathway of medical innovation: Improving upon already-effective treatments to make them easier on patients.
As is clear with the case of epinephrine for allergies, sometimes available treatments can work—but at a cost some can’t bear.
And yes, we’ve discussed the challenge of needles and supply issues, but we do also mean literal cost. In the U.S., a two-pack of generic epinephrine auto-injectors can cost patients several hundred dollars—and patients often need to replace these devices every year.
Of course, thigh injections aren’t even the most painful-yet-effective treatments out there that the medtech space is disrupting. In hearing about Neffy, we’re reminded of colonoscopy alternatives in the works. And we think of the patients and advocates calling for better options to replace VCUGs—invasive UTI screening procedures that have been shown to cause PTSD in children.
Sometimes, the most important innovation is not about providing a more effective device or treatment. It can be about easing the harm that accompanies treatment itself. Or which can keep patients from seeking treatment.
As medical innovators, we know patients deserve better. We hope the clinical trials ahead for neffy and similar products give them those options.