The Food and Drug Administration has not approved the first needle-free epinephrine nasal spray to replace epinephrine auto-injectors containing EpiPen.
The agency told drug company ARS Pharmaceuticals that it needed to conduct another study on the drug, called Nephy, to support approval, the company announced. stated in a statement Late Tuesday night.
The refusal came despite a resolution in May from the agency’s advisory committee recommending approval of the drug for children and adults. It is rare for the FDA to disapprove a drug recommended by a committee.
“We are extremely surprised by this action,” Richard Lowenthal, CEO of ARS Pharmaceuticals, said in a statement.
The company said it intends to appeal the FDA’s request for additional data.
Epinephrine is Used in the United States since 1901 It is highly effective in reversing anaphylaxis, the most severe type of allergic reaction. Anaphylaxis can occur within minutes of exposure to allergens such as peanuts or cat dander. Untreated, it can lead to death.
However, all currently available epinephrine treatment options require injections, which can be a problem for people who are afraid of needles.
Dr. Zachary Rubin, an allergist at Oak Brook Allergists in Illinois, called the FDA’s decision “shocking.”
“There is no alternative at this time,” said Rubin, who is not a member of the FDA’s advisory committee. “There’s basically an epinephrine auto-injector and a needle option, and people have been asking for years to have a needle-free option.”
A key concern at the May advisory committee meeting was the lack of clinical data, particularly the fact that the drug had not been studied in people experiencing anaphylaxis.
Ars Pharmaceuticals said the nasal spray is “comparable” to the EpiPen, based on studies conducted on animals as well as people without anaphylaxis.
Despite the concerns, the committee ultimately voted 16-6 in favor of the drug’s use in adults and 17-5 in favor of its use in children.
For Dr. Marian Amirshahi, a professor of emergency medicine at Georgetown University School of Medicine and a member of the advisory committee, the lack of data on anaphylaxis patients was a bottleneck. She voted against the drug both times.
“I listened to parents at the hearing talk about how traumatic it was for them to vaccinate their children,” Amirshahi said in an email to NBC News on Tuesday. Ta. “But for me as a parent, committee member, pharmacologist, and ER doctor who has seen my fair share of anaphylaxis, it’s not the injections that are scary, but the treatment of life-threatening conditions. It was the medicine that failed.”
Research into drugs for the emergency treatment of life-threatening allergic reactions raises difficult ethical questions.
To conduct a randomized controlled trial, adults and children suffering from severe allergic reactions would receive treatment or a placebo, an unethical scenario, Rubin said.
Amirshahi said the drug could be studied in people experiencing anaphylaxis in allergy departments and emergency rooms, where reliable treatments are available.
She said she hopes the FDA’s rejection will give her time to properly study the drug and see if it works.
“I think this drug has merit, but we don’t have enough efficacy data at this point,” she said.
The FDA requested additional studies comparing Nephi and epinephrine autoinjectors in people with allergen-induced nasal symptoms such as sneezing, itching, and nasal congestion.
The company said it plans to resubmit its application to the FDA in the first half of 2024.
