New non-opioid pain reliever moves closer to approval

Vertex Pharmaceuticals’ new painkiller significantly reduced moderate-to-severe pain in two phase 3 trials — suggesting it could be the new, non-addictive pain reliever we’ve been waiting for.

The challenge: Pain is the most common reason people seek out medical care, and while there are seemingly infinite causes of pain — from migraines and broken bones to infections and nerve disorders — we have very few options for treating it.

Over-the-counter meds, like ibuprofen and aspirin, are safe and easily accessible, but they are only effective for mild pain. Prescription opioids, like oxycodone and morphine, meanwhile, are highly effective, but they also cause a “high” that can be addictive and lead to misuse.

The new painkiller ​​led to a “clinically meaningful” reduction in pain in the 48 hours after the surgery.

Blocking pain’s pathway: A new painkiller that’s highly effective, but non-addictive is one of the holy grails of medicine, and the search for it has led Vertex Pharmaceuticals to a class of drugs called “sodium channel modulators.”

These medications act on a group of proteins that are involved in pain processing. Vertex is developing a drug, called VX-548, that it hopes will interfere with one of those proteins in a way that relieves pain, without any euphoric or addictive side effects.

What’s new? On January 30, Vertex shared the topline results of two phase 3 trials of its new painkiller as a treatment for moderate-to-severe acute pain (“acute” pain is usually defined as coming on suddenly, like from an injury or surgery, and lasting less than six months, while “chronic” pain develops gradually and lasts longer).

The trials involved a total of about 2,200 participants who were given either VX-548 or a placebo after undergoing abdominoplasty surgery (a “tummy tuck”) or surgery to remove a bunion from their foot. 

According to Vertex, the new painkiller ​​led to a “clinically meaningful” reduction in pain in the 48 hours after the surgery. It did not, however, meet its secondary endpoint: delivering more pain relief than a combination of the opioid drug hydrocodone (Vicodin) and acetaminophen (Tylenol).

Vertex also shared the results of a third phase 3 trial in which 256 people experiencing acute pain for a variety of reasons received VX-548 for up to 14 days. 

The drug was safe and well tolerated in that trial, leading to only mild-to-moderate adverse effects. About 83% of the participants rated the new painkiller as being “good,” “very good,” or “excellent” at treating their pain.

Looking ahead: There’s no word on when Vertex plans to publish the full trial results, but the company has said that it hopes to apply to the FDA by mid-2024 to get the medication approved to treat moderate-to-severe acute pain.

“For years, our goal has been to make a medicine with clinically meaningful pain relief for moderate-to-severe acute pain, with a safety and tolerability profile that’s better than an opioid,” David Altshuler, Vertex’s chief science officer, told BioPharma Drive. “We will file for approval with urgency.”

Vertex isn’t limiting itself to acute pain, though — an estimated one in five adult Americans lives with chronic pain, and a phase 3 trial of the new painkiller for long-lasting pain due to nerve damage from diabetes is ongoing.

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