A US drug company – ARS Pharma – which had been hoping for FDA approval last week of its flagship product, a needle-free treatment for adults and children with severe allergic reactions, has instead been asked for an additional study.
The company now plans to file a dispute resolution request with the FDA, and resubmit the medication in the first half of 2024, with hopes of getting approval by the end of next year.
STAT News reports that the product, called Neffy, is a nasal spray that delivers the same drug, epinephrine, as the auto-injectors currently used to treat anaphylaxis (a severe, whole-body allergic reaction to a chemical that has become an allergen).
Richard Lowenthal, CEO of ARS Pharma, said the company had been asked to conduct a repeat-dose study of Neffy, which he said was originally a post-marketing requirement.
Although an advisory committee had recommended the FDA approve the medication, some questioned whether ARS provided sufficient data on its efficacy compared with available epinephrine products.
Viatris, the maker of the EpiPen – and potentially the biggest competitor to Neffy – has also filed a petition asking for more data.
As of May, there were still questions about whether Neffy worked as well as auto-injector epinephrine, since much of ARS’ data were from people who were healthy or only had mild allergies (not anaphylaxis); there were limited safety data; no formal clinical efficacy trial; and results varied across trials.
The review process is based on surrogate measures of efficacy, such as how well Neffy raises a person’s blood pressure (a necessary step for calming an allergic reaction).
The study now required for Neffy’s approval will compare repeat doses of the drug with repeat doses of an epinephrine injection in people with allergen-induced allergic rhinitis.
Neffy is a smaller and more easily administered rival to auto-injectors like the EpiPen, which for years have been the only quick treatment option for people with severe allergies.
Epinephrine, which is the same thing as adrenaline, has been used for a century to treat anaphylaxis.
But existing treatments have run into several problems that the makers of Neffy hope to resolve, including supply and design.
Since 2017, there have been shortages of the EpiPen, and other auto-injectors have been pulled from the market over quality control problems.
Lowenthal said supply won’t be an issue; it’s the complicated design of the pens that has been a problem. Epinephrine itself is available, and Neffy will be using a simple spray device, the same one used for overdose reversal medication Narcan and several other products on the market.
Neffy’s nasal spray containers are about the size of a tea bag, and deliver a single dose into the nose with a one-handed pump.
Another issue is design. Beyond a fear of needles that might make users hesitate in an emergency, auto-injectors can be large and inconvenient to carry around, or tricky to use, leaving some unprepared when they go into anaphylactic shock.
ARS’ research suggests children as young as 12 can use Neffy by themselves (the drugmaker chose the name to be kid-friendly).
“It doesn’t matter if you dose it upside down, right side up, at an angle – it fires exactly the same way in every direction,” Lowenthal said. And ARS has designed a clip-on carrying case reminiscent of an AirPods case in which to keep the doses.
Another bonus is that Neffy can be stored at up to 50 degrees Celsius for up to three months, meaning users don’t need to worry about the drug going bad if they leave it in a hot car for an afternoon, pushing the temperature stability further than auto-injectors.
ARS is ready to have the product in pharmacies within eight weeks of approval, once the FDA issues its final labelling.
An epinephrine nasal spray has been difficult to develop because the drug is not well-absorbed through the nose, said Lowenthal, who has 25 years in drug development, having started his career as a new drug reviewer at the FDA.
ARS is also running trials to see if the medication can work for people with chronic hives or persistent asthma.
STAT News article – A needle-free anaphylaxis treatment runs into an FDA roadblock (Open access)
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