By Pat Anson, PNN Editor

An experimental non-opioid analgesic was effective in treating post-operative pain in two Phase 3 clinical trials, setting the stage for a New Drug Application to the FDA by Vertex Pharmaceuticals later this year.

The drug – called VX-548 – was given orally to patients with moderate-to-severe pain in the first 48 hours after an abdominoplasty or bunionectomy, two minimally invasive surgeries. VX-548 was more effective in reducing pain than the placebo, but it was not more effective than a combination of the opioid hydrocodone and acetaminophen, more commonly known as Vicodin.

In a third Phase 3 study, VX-548 was effective for up to 14 days across a broad range of surgical and non-surgical acute pain conditions. Over 80% of patients in that study rated VX-548 as good, very good, or excellent in treating pain.

“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. The VX-548 benefit-risk profile ideally positions it to potentially fill the gap between medicines with good tolerability but limited efficacy and opioid medicines with therapeutic efficacy but known risks, including addictive potential,” Reshma Kewalramani, MD, CEO and President of Vertex, said in a press release.

“With FDA Breakthrough and Fast Track Designations in hand, we are working with urgency to file the New Drug Application for VX-548 and bring this non-opioid medicine to the millions of patients who suffer from acute pain each year in the U.S.”

VX-548 was generally safe and well tolerated in all three Phase 3 studies. Most of the adverse events reported by patients were mild to moderate, such as nausea and constipation.

Unlike opioids, VX-548 blocks pain in the peripheral nervous system, rather than the brain. That means it won’t have euphoric effects or be addictive. If approved for use by the FDA, experts say it would give patients and providers a much-needed alternative to opioids.

“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 Jessica Oswald, MD, a Vertex consultant and Associate Physician in Emergency Medicine and Pain Medicine at University of California San Diego.

“The Phase 3 safety and efficacy across the three studies are impressive and demonstrate VX-548’s potential to change the paradigm of pain management. I look forward to the potential of having a new class of acute pain medicine — the first in more than two decades — to use as an alternative to opioids to help the millions of people impacted by acute pain.”

The risk of a surgery patient misusing opioids or becoming addicted is actually quite low – less than one percent. One recent study even found that restricting the use of opioids during surgery leads to more post-operative pain.

Vertex hopes to have VX-548 approved for a wide variety of pain conditions, not just post-operative pain.  Last month, the company released positive results for the drug in reducing pain from diabetic peripheral neuropathy.

Vertex also recently won approval from the FDA for its CRISPR gene cell therapy for sickle cell disease, which potentially offers a cure for the painful blood disorder.