Story by Jennifer Calfas
Feb 13, 2024
People who are suffering from severe pain but don’t want to risk addiction to an opioid are closer to a new option for treatment.
People who are suffering from severe pain but don’t want to risk addiction to an opioid are closer to a new option for treatment.
Vertex Pharmaceuticals on Tuesday reported positive study results for its closely watched non-opioid painkiller. The drug lowered the moderate-to-severe acute pain reported by study volunteers, a sign it could be the first in a new class of painkiller to be approved for use.
But the experimental medicine is more likely to provide an alternative to opioids, rather than supplant them, because it didn’t work better than a widely used opioid drug sold under the brand name Vicodin.
Vertex said it would file for approval from the U.S. Food and Drug Administration by the middle of this year.
“If and when this medicine is approved, it will offer a new option for patients who need pain relief but who do not want to take an opioid,” Vertex Chief Scientific Officer David Altshuler said in an interview.
Vertex released the results in a press release, not a peer-reviewed medical journal. The company said the researchers who conducted the studies would publish their results in a journal later this year.
About 80 million Americans are prescribed a medicine for moderate-to-severe acute pain each year, according to Vertex. They can choose from a variety of treatments. Many of them take opioids because they work so well.
Yet opioids are highly addictive, and their widespread use has fueled an epidemic of addiction and overdose deaths in the U.S.
To avoid addiction, many doctors, patients and health authorities have sought an alternative that provides as much relief from pain without creating the dependence on use.
Vertex’s drug, code-named VX-548, belongs to a new class of medicines that targets molecular mechanisms involved in feeling pain. Researchers have pursued this so-called NaV class for years but experienced repeat setbacks.
NaV refers to the channels through which sodium flows in and out of cells. VX-548 blocks the flow of sodium through a channel called NaV1.8, which plays a role transmitting pain signals to the brain.
The drug, in other words, works by blocking or diminishing pain at the source before it heads to the brain and creates the feeling of pain.
Boston-based Vertex studied VX-548 in three late-stage, or Phase 3, trials that enrolled more than 2,400 subjects with conditions causing moderate-to-severe acute pain.
Two of the trials compared the efficacy of VX-548 against a placebo and Vicodin among subjects who either underwent a “tummy tuck,” or abdominoplasty, surgery or a bunion surgery.
The third study measured its efficacy among a range of surgical and nonsurgical acute pain issues.
Study subjects didn’t show any serious adverse effects in the three studies, the company said.
RBC Capital analyst Brian Abrahams said VX-548’s profile suggested it would be a “niche player and not a game changer” in treating acute pain.
The drug “is likely to be a viable alternative for certain patients who would not otherwise be able to take NSAIDs or might be at risk for addiction with opioids, but not something that will completely upend the treatment paradigm,” he said.
Vertex is best known for its blockbuster cystic fibrosis treatments. The company received the first U.S. approval last month for a therapy that uses Crispr gene-editing technology; the drug, called Casgevy, treats people with sickle-cell disease.
VX-548 could open a new, potentially multibillion franchise in pain for the biotech company, Evercore ISI analyst Liisa Bayko said. “It’s a totally new class of drugs that have never made it this far,” she said in an interview before the company released the trial results.
If approved, however, the drug will face heavy competition, including from low-price generics, according to analysts. To gain traction, Vertex will have to build experience and relationships in the market.
Stuart Arbuckle, Vertex’s chief operating officer, said the company aims to sell this new class of medications across different pain types. Last month, the company released positive results for the drug reducing pain caused by diabetes-related nerve damage.
Write to Jennifer Calfas at jennifer.calfas@wsj.com