Experimental Drug Cuts Off Pain at the Source, Company Says

Vertex Pharmaceuticals of Boston announced Tuesday that it had developed an experimental drug that relieves moderate to severe pain, blocking pain signals before they can get to the brain. It works only on peripheral nerves — those outside the brain and the spinal cord — making it unlike opioids. Vertex says its new drug is expected to avoid opioids’ potential to lead to addiction.

The company reported that it had completed two randomized studies, the first in 1,118 people who had abdominoplasties and the other in 1,073 people who had bunion surgery. The two procedures are commonly used in studies of people with acute pain, the temporary kind that is brought on by something like a surgical procedure and is likely to ease with time.

In its clinical trials, Vertex measured the drug’s effect with a standard pain scale in which patients rated pain severity from 1 to 10, with 10 the most severe. Those taking its drug had a statistically and clinically meaningful reduction in pain, it reports. A third study looked at safety and tolerability of the drug in people experiencing pain from a variety of conditions.

Buoyed by the results, which are yet to be published or presented at a meeting, Vertex plans to apply to the Food and Drug Administration by midyear for approval to market the drug, a pill that, for now, is called VX-548.

“This has the potential to be a blockbuster,” said Dr. Stephen Waxman, a professor of neurology, neuroscience and pharmacology at Yale. Dr. Waxman was not associated with the study but was paid a speaking honorarium by the company. He predicted that the Vertex drug would be only the first foray into this new area.

“I like to think it’s the beginning of nonaddictive medicines for pain,” he said.

For now, most people needing relief from moderate to severe pain have two options: drugs like ibuprofen and COX-2 inhibitors, or opioids. The drugs like ibuprofen are not very effective, and the opioids, as is well known, can be addictive because of the way they work. There is no way to separate the effects of opioids — pain relief — from the side effects: changes in thinking, cognition, energy and emotions.