FDA approves Journavx, new non-opioid painkiller from Vertex: ‘An important public health milestone'

Boston Globe

30-01-2025

In two late-stage trials of patients who underwent tummy tuck surgery or removal of a bunion, Journavx led to a 50 percent reduction in pain, Vertex said. It acted faster than a placebo and with fewer side effects than a pill that combined acetaminophen and the opioid hydrocodone, the active ingredients in Vicodin.
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Significantly, Journavx didn't provide better pain relief than the opioid combination in one of the trials and was inferior to the combination in the other trial. Nonetheless, Vertex executives say Journavx's ability to soothe pain without the risk of addiction will likely make it a blockbuster, a drug that generates sales of at least $1 billion a year.
'We think this is the sort of product that, frankly, physicians and patients have been waiting for,' said Stuart Arbuckle, Vertex's chief operating officer.
The most common side effects in patients who received Journavx in the clinical trials included itching, muscle spasms, and rashes.
The approval comes as the nation continues to grapple with a decades-old opioid crisis. More than 81,000 people died of opioid overdoses in the United States in 2023, according to the National Center for Health Statistics.
Doctors prescribe opioids for acute pain to 40 million Americans a year, and about 85,000 of them develop opioid use disorder, according to Vertex.
The company has also tested Journavx as a treatment for chronic pain, a bigger market than acute pain, but the results of clinical trials so far have been underwhelming.
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Dr. Jessica McCoun, an Atlanta-based anesthesiologist and principal investigator in the trials, said she considers the new Vertex drug 'a very big tool in the toolbox' for treating pain. 'The more tools we have that we can combine together to treat a patient effectively ... That's the best we can do,' she said.
A bottle of Journavx
Courtesy of Vertex
Among the patients who received Journavx in the clinical trials was a 50-year-old woman from the Atlanta area. She had a nose job in October 2023 and began taking the pill immediately afterward in a third Vertex trial for people who underwent a variety of surgeries.
The woman, who only wanted to be identified by her first name, Samantha, because of the sensitivity of the subject, said she took Journavx for four days. She never felt any pain, she said, even though her operation was a 'pretty severe reconstruction.'
'For the level of pain I should have been in, this medicine was very, very effective,' she said. 'I think it's spectacular.'
Some experts were more cautious in their assessments.
Dr. Adriane Fugh-Berman, a professor of pharmacology and physiology at Georgetown University Medical Center, welcomed the FDA approval. But she contended it was premature to declare that Journavx posed no risk for addiction, saying it wasn't tested long enough in clinical trials.
'It's definitely a promising painkiller, but I haven't seen studies that lasted longer than a couple of weeks in humans,' said Fugh-Berman.
Drugmakers, she went on, have made similar claims for other medicines and turned out to be wrong. She cited gabapentin, an anti-seizure medication used for nerve pain that experts say can lead to abuse and dependency.
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Two market analysts had predicted the FDA would approve the drug based on the results of clinical trials, but they expressed different levels of enthusiasm.
David Risinger, an analyst for the Boston investment bank Leerink Partners, called the clinical trial results 'compelling' and predicted that the medicine will generate peak annual sales of $4 billion in the 2030s.
'We think physicians and insurance companies would rather send patients home with a non-opioid pill prescription rather than an opioid pill prescription,' he said. 'It's a huge market opportunity.'
Brian Skorney, an analyst with Baird, also said Journavx's approval was 'as close to a sure thing as one can get' because it reduced pain when compared to a placebo, the chief goal of the studies.
But he said Vertex put its thumb on the scale for a secondary goal of the trials by comparing the drug to the equivalent of a low-dose Vicodin rather than to more potent opioids that doctors sometimes prescribe. He said it would have been more meaningful had Vertex simply compared Journavx to inexpensive over-the-counter painkillers that people have in their medicine cabinets, such as ibuprofen.
'I think people will be disappointed with, ultimately, how much it sells,' he said of Journavx.
Vertex, however, seems to be confident and added 150 sales employees last year in anticipation of the FDA approval. The company has about 6,100 employees worldwide, including 4,200 in Massachusetts.
Despite how common pain is, there hasn't been a significant innovation for treating it in decades. Aspirin dates to the late 19th century, while acetaminophen, ibuprofen, and naproxen sodium
were invented generations ago.
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Opioids date back thousands of years. But they have led to a devastating public health crisis, fueled in no small part by OxyContin, the opioid tablet made by Purdue Pharma.
For years, multiple companies, including Cambridge-based Biogen, the South San Francisco biotech Genentech (which was later acquired by Roche), and the Swiss drug giant Novartis have tried unsuccessfully to develop a new class of painkillers. In 2022, Regeneron Pharmaceuticals, of Tarrytown, N.Y., shelved an experimental drug for osteoarthritis and chronic low back pain that it had long worked on after clinical trials raised safety concerns.
Painkillers today fall into two major classes: anti-inflammatory analgesics, such as aspirin and ibuprofen, and opioids. Anti-inflammatories work by reducing swelling at the location of pain. Opioids change the brain's perception of pain, mimicking the natural pain-relieving chemicals known as endorphins produced in the brain.
In contrast, Vertex's new drug interrupts pain signals before they reach the brain, blocking certain proteins called sodium channels that transmit pain in the peripheral nervous system. Because the non-opioid drug bypasses the central nervous system, the company says, it provides pain relief without the risk of addiction.
Globe correspondent Stella Tannenbaum contributed to this report.
Jonathan Saltzman can be reached at