The U.S. Food and Drug Administration (FDA) granted approval on Thursday for suzetrigine, marking the first novel pain relief medication to enter the market in more than two decades.
Suzetrigine, a prescription pill available in 50-milligram doses, is taken every 12 hours following an initial higher dose. It will be marketed under the brand name Journavx.
“A new non-opioid analgesic therapeutic class for acute pain offers an opportunity to mitigate certain risks associated with using an opioid for pain and provides patients with another treatment option,” stated Dr. Jacqueline Corrigan-Curay, acting director of the FDA’s Center for Drug Evaluation and Research, in a news release. She further emphasized, “This action and the agency’s designations to expedite the drug’s development and review underscore FDA’s commitment to approving safe and effective alternatives to opioids for pain management.”
Pain medications, known as analgesics, are among the most frequently prescribed drugs in hospitals, according to government surveys. Every year, approximately 80 million Americans receive prescriptions for pain treatment, with nearly half of those prescriptions being opioids, which carry risks of addiction and dependency. This data comes from a study conducted by Vertex Pharmaceuticals, the company behind suzetrigine.
Suzetrigine is the first new pain relief medication approved in the U.S. since Celebrex, a Cox-2 inhibitor classified as a nonsteroidal anti-inflammatory drug, which gained FDA approval in 1998.
Dr. Sergio Bergese, an anesthesiologist at Stony Brook University’s Renaissance School of Medicine, explained that pain perception involves multiple areas of the body. When tissue damage occurs, nerve cells send electrical signals to the brain, which interprets them as pain.
While opioids function by dulling pain perception in the brain, suzetrigine operates differently by preventing pain-signaling nerves from transmitting signals in the first place. “This drug, what it is doing is interrupting that path, so even though the tissue injury exists, the brain doesn’t know,” Bergese explained.
Significantly, suzetrigine does not induce the euphoria or high associated with opioids, making it unlikely to lead to dependence or addiction.
The drug’s origins trace back to a unique genetic discovery in Pakistan, where researchers found a family capable of walking over hot coals without experiencing pain. Scientists discovered that members of this family lacked a specific gene responsible for transmitting pain signals in the skin.
“They knew that they were on something hot; they knew they could feel the coals. So it’s not impacting the nerves that do heat and touch and stuff like that. It is just these pain-conducting nerves,” explained Stuart Arbuckle, chief operating officer of Vertex Pharmaceuticals. “They were, in every other way, normal.”
Despite the discovery, it took scientists 25 years to harness this biological mechanism and translate it into a pharmaceutical solution.
Dr. Stephen Waxman, who directs the Center for Neuroscience and Regeneration Research at the Yale School of Medicine, explained the science behind pain signaling. “Neurons talk to each other by producing series of nerve impulses, like a Morse code,” he said. “And nerve impulses are produced by tiny molecular batteries within the membranes of neurons. The molecular batteries are called sodium channels.”
Suzetrigine functions by blocking a specific sodium channel responsible for transmitting pain signals.
The journey to developing a targeted sodium-channel blocker was fraught with challenges, as previous attempts had failed to isolate a single channel that could be safely inhibited. However, with suzetrigine’s approval, Waxman believes this breakthrough will pave the way for even more effective pain medications. “It is an important step forward, because it provides proof of concept that a [sodium-channel blocker] can reduce pain in humans,” he said. “That opens up the door to a second generation of even more effective [medications].”
Suzetrigine is administered in two dosages. Clinical trials involved an initial 100-milligram dose, followed by 50-milligram doses taken every 12 hours.
Physicians caution that the medication may not be effective for every individual or type of pain.
In two studies involving nearly 600 participants, suzetrigine demonstrated effectiveness in managing post-surgical pain following abdominal and foot operations. Its pain relief capabilities were comparable to Vicodin, a combination of acetaminophen and the opioid hydrocodone. However, researchers did not directly compare suzetrigine to Vicodin, making it difficult to determine which drug was superior.
Participants in the studies initially reported pain levels averaging around seven on a scale of zero to ten. With suzetrigine, this rating dropped by approximately 3.5 points.
“It’s not like eliminating all pain,” Arbuckle acknowledged. “It’s reducing pain by about 50%.”
A separate study examined the drug’s effects on individuals experiencing back pain due to sciatica. In this case, suzetrigine reduced pain levels by about two points, which was the same improvement reported by those given a placebo. These results suggest that suzetrigine may not be particularly effective for chronic pain conditions.
Vertex Pharmaceuticals disputes this conclusion, arguing that the drug has shown promise in treating various types of chronic pain. The company is currently conducting further trials on patients with diabetic neuropathy—a condition where prolonged high blood sugar damages nerves, causing pain, numbness, tingling, and muscle weakness.
The sciatica study had a smaller sample size compared to the other trials, with about 100 participants receiving either suzetrigine or a placebo. Due to the limited sample, researchers acknowledge that there may not have been enough participants to detect a significant difference between the drug and the placebo. Additionally, placebo effects tend to be pronounced in pain studies, complicating the interpretation of results.
“In our opinion, the drug did what we expected to do in terms of the amount of pain relief,” Arbuckle said. “But unfortunately, as often happens in studies in pain, there is quite a large placebo response.”
Medical professionals specializing in pain management have welcomed the approval of suzetrigine, recognizing its potential as an alternative to opioids.
“The more options we have, the better we’re able to treat each and every patient,” said Dr. Kimberley Mauer, an anesthesiologist at Oregon Health and Science University.
However, Mauer pointed out that the drug’s accessibility may be influenced by its cost. Vertex Pharmaceuticals has set the wholesale price at $15.50 per 50-milligram pill, but the company has assured that financial assistance programs will be available for eligible patients.
The ultimate impact of cost on accessibility remains uncertain, as insurance companies will play a role in determining coverage options.
“It might limit some patients getting it. So we just have to kind of see, and it’s hard to tell until it kind of gets out on the market,” Mauer said.