The Food and Drug Administration didn’t approve an epinephrine nasal spray that might have been the primary needle-free alternative to epinephrine autoinjectors, including EpiPens.Â
The agency told drugmaker ARS Pharmaceuticals that it needed to conduct one other study on the drug, called Neffy, to support approval, the corporate said in a press release late Tuesday night.
The rejection got here despite the agency’s advisory committee in May voting to recommend approval of the drug in children and adults. It’s rare that the FDA doesn’t approve drugs really helpful by its committees.Â
“We’re very surprised by this motion,” Richard Lowenthal, the CEO of ARS Pharmaceuticals said within the statement.
The corporate said it will appeal the FDA’s request for extra data.
Epinephrine has been utilized in the U.S. since 1901 and is extremely effective in reversing anaphylaxis, essentially the most severe kind of allergic response. Anaphylaxis can occur inside minutes of being exposed to an allergen, resembling a peanut or cat dander. Without treatment, it might be deadly.
All currently available epinephrine treatment options, nevertheless, have to be injected, which is usually a problem for people who find themselves afraid of needles.
“I’m shocked,” Dr. Zachary Rubin, an allergist at Oak Brook Allergists in Illinois, said of the FDA’s decision.
“There aren’t any alternatives without delay,” said Rubin, who is just not a member of the FDA’s advisory committee. “You principally have epinephrine autoinjector devices, needle options, and folks have been clamoring for years to get a needle-free option.”
A serious point of concern in the course of the May advisory committee meeting was a scarcity of clinical data, particularly the undeniable fact that the drug was not studied in people experiencing anaphylaxis.
ARS Pharmaceuticals said that the nasal spray was “comparable” to an EpiPen, based on studies conducted in animals in addition to in people without anaphylaxis.
Despite its concerns, the panel ultimately voted 16-6 in favor of the drug to be used adults, and 17-5 in favor of the drug use in children.
For Dr. Maryann Amirshahi, a professor of emergency medicine at Georgetown University School of Medicine and a member of the advisory committee, the dearth of knowledge in patients with anaphylaxis was a sticking point. She voted against the drug each times.
“I listened to the parents within the hearing and I heard them speak about how traumatic it was to present their children a shot,” Amirshahi said in an email to NBC News on Tuesday. “But to me, as a parent, panel member, pharmacologist, and ER doc who has seen her fair proportion of anaphylaxis, the scary thing was not the shot, but a drug failing in treating a life-threatening condition.”
Studying a drug meant to be an emergency treatment for life-threatening allergic reactions raises thorny ethical questions.
So as to do a randomized controlled trial, adults and kids affected by a severe allergic response would either receive the treatment or a placebo, a scenario that might be unethical, Rubin said.
Amirshahi said that it is feasible drug might be studied in people experiencing anaphylaxis in an allergist’s office or an emergency room where there can be a fail-safe treatment readily available.
She said that she’s hopeful that the FDA’s rejection will allow for time to check the drug properly and make certain it’s effective.
“While I believe the drug has merit, it doesn’t have adequate efficacy data at this point,” she said.
The FDA has requested an extra study comparing Neffy to an epinephrine autoinjector in individuals with allergen-induced nasal symptoms including sneezing, itching and congestion.
The drugmaker said it plans to resubmit its application to the FDA in the primary half of 2024.
