OTC naloxone? It's possible

FDA officials are considering whether naloxone should be more widely available beyond medical settings, including through over-the-counter (OTC) sales and/or an intranasal version of the drug.

In an April scientific workshop at FDA headquarters, representatives of community overdose prevention programs and others called on the agency to take rapid action in light of a growing epidemic of U.S. overdose deaths, a trend largely driven by misuse and abuse of prescription and illicit opioid drugs.

The injectable, µ-opioid receptor antagonist naloxone is rated very effective against opioid overdose, with few serious side effects.

The Harm Reduction Coalition stated that since 1996, a number of local programs such as needle-exchange efforts have distributed naloxone and provided education to drug users and people around them so that the medication can be administered if an overdose is observed. CDC recently published a survey from the coalition showing that 48 of those programs said that from 1996 to 2010, they received reports of a total of 10,171 overdose reversals. However, according to the report, of the 25 states with higher than median drug overdose deaths in 2008, 19 had no community program that distributed naloxone.

Douglas Throckmorton, MD, deputy director for regulatory programs, FDA's Center for Drug Evaluation and Research, told the meeting, "We understand that we bear a part in this. We need to be part of the solution to addressing prescription drug abuse." The suggestions from the state and local efforts, which might be considered pilot programs, seem to be bearing fruit, he said.

"Certainly, considering naloxone as an over-the-counter drug is forging new territory," said Andrea Leonard-Segal, MD, director, FDA Division of Nonprescription Clinical Evaluation, Office of Nonprescription Products. Classifying the drug OTC would probably be accomplished through the new drug application (NDA) process, she said. To accomplish it by FDA rulemaking would take years. The NDA process for switching to OTC classification, she said, would require a fresh look at the drug's chemistry, pharmacology/toxicology, microbiology, and clinical pharmacology. There might not be a need for efficacy data if a current formulation were used, she said.

If there were a new formulation, such as topical or intranasal, the agency would need new clinical safety data. For example, she said, "If the product were more bioavailable than the reference drug to which it was compared, it probably would be wise to market it first by prescription to acquire a post-marketing safety database to support OTC use." Another question would be whether the drug might be converted into an opioid that could be abused, she said.