The Food and Drug Administration on Tuesday did not approve drug company ARS Pharmaceuticals’ epinephrine nasal spray Nephy, making it the first needle-free option for Americans to treat severe allergic reactions until more trial data are available. remains off the market.
ARS expected the FDA to approve Nephy for use in adults and children weighing more than 30 kilograms. Like the EpiPen and others, the spray required a prescription. epinephrine injection currently used to treat anaphylaxis.
Epinephrine is very important for treating potentially life-threatening emergencies allergic reaction. If this new spray is ultimately approved, it will be a welcome alternative for many families with children with severe allergies who want to avoid needles.
FDA regulators determined Tuesday that more data is needed to evaluate the safety of repeated doses of the nasal spray before it can be approved, according to ARS. The drugmaker expressed disappointment with Tuesday’s decision, as some FDA advisers had suggested at a previous meeting in May that there was no need to collect additional data in post-market trials for the drug.
“We are very surprised by this action and the current delayed requirement to change repeated-dose studies from post-market requirements, which were previously coordinated with the FDA, to pre-approval requirements, especially given the positive recommendations. “The committee’s vote is positive,” Richard Lowenthal, co-founder, president and CEO of ARS Pharma, said in a statement late Tuesday. “With this measure, Nephi For millions of people who are at risk of severe, potentially life-threatening allergic reactions.Both patients and caregivers are welcome. NephiAnd we aim to complete newly requested trials as quickly as possible to meet patient needs. ”
“The rapid use of epinephrine in the community is limited by significant barriers. Many patients are afraid of needles and many are not accustomed to self-injecting. Also, carrying the currently available equipment is difficult. It’s unrealistic,” Lowenthal told the FDA’s panel of outside advisers. at the May meeting Weigh the product.
Lowenthal told the committee that the company plans to offer the product in a “slim Nephi carrying case” that can hold two sprayers.
According to Lowenthal, 85% of patients facing severe allergies would be willing to carry epinephrine with them every day if Neffy was an option, up from about 55% with current options. Lowenthal said.
“If they don’t have it, that’s moot. They don’t give birth, they don’t have medicine, they go to the hospital,” Lowenthal said in May.
Pharmaceutical company To tell Neffy nasal spray works by administering epinephrine to patients facing an allergy attack using two technologies already used in other FDA-approved products. One is a substance called dodecylmaltoside, licensed from the pharmaceutical company Neurelis, which the company claims “facilitates drug absorption” through the mucous membrane, the cells that line the airways from the nose to the lungs. Neurelis uses this ingredient and is branded as: Intravailwith its own spray to treat epilepsy.
the other one is, sprayer A device sold by Aptar Pharma and already used in many products. This is the same type of sprayer used in the. Narcan brand naloxone nasal sprayused to treat drug overdoses, Recently got the green light Cleared for over-the-counter sale by the FDA.
Despite the availability of several generic alternatives, some Americans continue to face high prices to purchase currently available epinephrine injection devices each year.Brand-name EpiPen and its generic alternatives could be costly for some households Over $200 per yearwhich causes some states to explore the cap About the price of epinephrine.
More epinephrine nasal sprays are also in development.Advertised by pharmaceutical company Brin Pharma promising results Utuly, an epinephrine nasal spray being tested, may have better performance than current syringes.
Questions about effectiveness
The FDA’s request for more data only delayed ARS’ efforts to bring the drug to market.
The company previously said the FDA may decide to approve Nephi. By mid-2023. However, ARS revealed in June that the FDA had told the company that an approval decision would take until September. The company said it needed extra time to complete “discussions regarding labeling and postmarketing commitments” that were raised after an FDA advisory committee meeting on Nephi.
a majority Members of the FDA’s Lung Allergy Drugs Advisory Committee have wrestled with many questions about whether the spray might be less effective than current injectables, and ultimately support the product’s benefits outweighing the risks. Voted.
“Following strong support for our clinical data, Nephi “At the PADAC meeting in May, there was limited time to answer the final questions and complete the labeling,” Lowenthal said. release.
Although epinephrine is widely accepted by experts as effective in treating severe allergic reactions, the use of the chemical, which is purified adrenaline, Its origins date back to the early 20th century — predates the existence of the FDA. This means that the initial use of epinephrine to treat anaphylaxis will require clinical trials to directly prove its effectiveness, as well as a better understanding of what measurements of the drug in the body are needed for it to be effective. This means that it was born without undergoing any clinical trials.
The original EpiPen was approved in 1987 without clinical trials or detailed data on how the drug is absorbed or how it affects the body, the FDA said. To tell.
Clinical trials proving that administering epinephrine as a spray as well as an injection would be ethically difficult to conduct, FDA officials acknowledged.
Instead, ARS Pharmaceuticals various studies To generate so-called “surrogate” data aimed at figuring out whether the spray is as effective as the injection.
These included dosing subjects exposed to seasonal allergens and measuring drug concentrations in the body, which produced some conflicting data. Comparisons with measurements from epinephrine injections also yielded mixed results.
“I really want this product to work. I mean, it definitely benefits from a needle-free means of delivering epi. And I think the sponsors have done a great job of finding that balance.” I think so. But I think we’re using it,” FDA committee member Lewis Nelson said at a May meeting.
Nelson, like other committee members, praised the company and the FDA. Approach to solution Some of the large variations seen with other epinephrine injections. But he also said he believes more data will be needed before the drug can be approved.
“In the absence of better clinical data, we really don’t want to recommend approval of a product based on inconsistent and somewhat confusing surrogate data and end up harming patients because of it,” he said at the conference. No,” he said.
