Vertex Pharmaceuticals said Tuesday that its experimental painkiller was as effective treating acute postsurgical pain in clinical trials as a commonly prescribed pain reliever containing an opioid — without the risk of addiction.
The Boston drug maker plans to apply by the middle of the year to the Food and Drug Administration to approve the medicine, currently known by its lab name VX-548. If approved, it would represent the first new class of painkillers in decades and could — along with other pain relievers Vertex has in its pipeline — generate more than $10 billion a year for the company, according to one analyst.
Although the novel pill didn’t outperform an opioid painkiller, Vertex contends that it warrants approval because it doesn’t pose the same potential for addiction.
“We are very pleased with the results from the VX-548 pivotal program, which demonstrate a compelling and consistent combination of efficacy and safety across multiple acute pain conditions and settings,” Reshma Kewalramani, Vertex’s chief executive and president, said in a statement.
Vertex tested the drug in two late-stage trials on nearly 2,200 patients between the ages of 18 and 80 who underwent tummy tuck surgery or bunion removal. The patients were randomly divided into three groups and received VX-548, a pill containing the opioid hydrocodone plus acetaminophen, or a placebo.
Regardless of which surgical procedure patients underwent, the experimental drug resulted in a “statistically significant improvement” that rivaled the benefits of the opioid medicine over 48 hours.
The drug was also safe and effective for up to 14 days when tested in a third late-stage trial in 256 patients with acute pain stemming from surgical and nonsurgical causes, everything from a shoulder operation to severe bruises.
“As a physician treating patients suffering from pain for many years, I know firsthand the critical need for new, efficacious, and safe treatment options,” said Dr. Jessica Oswald, a pain specialist at the University of California San Diego and a paid investigator in the trials. “I look forward to the potential of having a new class of acute pain medicine . . . to use as an alternative to opioids to help the millions of people impacted by acute pain.”
Joseph Stringer, an analyst for the Boston investment bank Needham & Company, said in a note to investors that he considered the trial results “slightly below expectations” because VX-548 didn’t prove superior to the opioid medicine, even though it’s not addictive. He expected the FDA will likely approve the new drug but said commercial “uptake could be a challenge” because the opioid painkiller is generic and likely to be much cheaper.
Vertex, best known for its blockbuster cystic fibrosis drugs, has worked for several years on painkillers that would provide the relief of opioids without the potential for addiction. More than 131 million opioid prescriptions were filled in the United States in 2022 despite an epidemic of addiction and overdoses, according to the Centers for Disease Control and Prevention.
Pain is ubiquitous, but there hasn’t been a major innovation for treating it in decades. Aspirin dates to the late 19th century, while acetaminophen, ibuprofen, and naproxen sodium were invented generations ago.
Opioids date back thousands of years and are highly effective, particularly against severe pain. But they have led to a decadeslong public health crisis, fueled in no small part by OxyContin, the opioid tablet made by the disgraced firm Purdue Pharma.
For years, multiple companies, including Cambridge’s Biogen, the South San Francisco biotech Genentech (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., abandoned an experimental drug for osteoarthritis and chronic low back pain that it had long worked on after clinical trials raised safety concerns.
Painkillers 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.
Vertex’s drug, if approved, would represent a third class. Vertex wants to interrupt pain signals before they reach the brain, blocking certain proteins called sodium channels that transmit pain in the peripheral nervous system. Because VX-548 bypasses the central nervous system, it provides pain relief without the risk of addiction, according to Vertex.
On Dec. 13, the company reported that its experimental painkiller significantly decreased chronic nerve pain caused by diabetes in a small, midstage trial of 192 patients. After 12 weeks, patients who rated pain on a scale of zero (no pain) to 10 (worst pain imaginable), showed reductions in pain levels of more than 2 points — a statistically significant decline.
In addition, more than 30 percent of patients reported a 50 percent reduction in pain regardless of whether they had received a low, medium, or high dose. More than 20 percent of patients who received high and middle doses said their pain plunged by at least 70 percent.
When Vertex released the encouraging results in December, its shares soared 13 percent, catapulting the market value of the biotech past $100 billion.
Vertex is now testing the drug on patients with chronic pain in a large late-stage trial. The application to the FDA in mid-2024 would seek approval only for acute pain because the results of the bigger study in chronic pain aren’t expected until later.
Jonathan Saltzman can be reached at firstname.lastname@example.org.