The Food and Drug Administration declined to approve an epinephrine nasal spray that could have been the first needle-free alternative to the EpiPen, the manufacturer said.
Instead, ARS Pharmaceuticals said late Tuesday the FDA is now requesting more studies prior to approval of its product, called Neffy.
Epinephrine has been used since 1901 and is the first line of defense to treat allergic reactions, including anaphylaxis. But all current epinephrine treatments, like the well-known EpiPen and other autoinjectors, use needles — which poses a problem if people are needle-phobic.
The rejection came despite an agency advisory committee in May recommending the FDA approve the product for children and adults suffering severe allergic reactions, without the need for additional studies to demonstrate its efficacy or safety.
“We are very surprised by this action,” said Richard Lowenthal, president and CEO of ARS Pharmaceuticals. He said the FDA is asking the company to conduct a repeat-dose study before approval, something that was originally supposed to be a post-marketing requirement.
The FDA wants a study that will compare repeat doses of Neffy to repeat doses of epinephrine injection in people with allergen-induced symptoms, including sneezing, itching and congestion.
“We are deeply disappointed that this action further delays the availability of Neffy for the millions of people who are at risk of a potentially life-threatening severe allergic reaction,” Lowenthal said. “Patients and caregivers are waiting for Neffy, and we aim to complete the newly requested trial as quickly as possible to meet the needs of patients.”
Aside from being needle-free, part of the appeal of Neffy was that it could provide competition to the expensive EpiPen.
In 2007, a pack of two injectors cost $100, but when Mylan bought the product, it hiked the price to more than $600 by 2016 — despite each pen costing a fraction of the to make.
As a result of the outcry, Mylan began selling an “authorized generic” for $300. An authorized generic is essentially the same product as the brand-name drug, just marketed as a generic.
Mylan eventually merged with a division of Pfizer to form Vitaris, the current manufacturer of EpiPen.
The FDA advisory panel in May voted 16-6 in favor of the drug for adults and 17-5 in favor for children for the treatment of severe allergic reactions, including anaphylaxis.
But the agency was concerned about a lack of clinical data, even though the product could be lifesaving.
The manufacturer compared its product to already approved injectable epinephrine products, rather than conduct large clinical trials. It wasn’t studied in people experiencing anaphylaxis.
Lowenthal said the company plans to file a dispute resolution request with the FDA to appeal the additional trial request, and anticipates a resubmission to the FDA in the first half of 2024, with the hope of approval by the end of next year
