By Sriparna Roy and Khushi Mandowara
(Reuters) -Vertex Pharmaceuticals' experimental drug reduced acute, post-surgical pain without the risk of addiction, a major milestone in the company's decades-long efforts to bring a non-opioid pain medicine to market.
The company plans to file for U.S. approval by mid-2024 for the drug, which works by blocking pain signals at its origin before they reach the brain. If approved, it could achieve annual sales of more than $5 billion, according to analysts.
When compared to a placebo, the drug was more effective in reducing the intensity of pain after 48 hours in two late-stage studies, Vertex (NASDAQ:VRTX) said on Tuesday.
The treatment, however, failed to meet the secondary goal of reducing pain when compared to a combination of the opioid drug hydrocodone and acetaminophen.
Shares of the drugmaker, up more than 35% in the past 12 months, slipped to $433.75 in early trading.
Still, analysts hailed the results as a win for Vertex and said the drug was likely to gain approval for treatment of acute pain, offering a much-needed alternative for addictive opioid painkillers that have fueled a national crisis.
"Investors and the company did not expect superiority over opioid as there is a huge safety (and) addiction advantage even without that," said Wells Fargo analyst Mohit Bansal.
While Vertex's drug works by blocking pain signals at source, opioids trigger the brain's reward centers as they travel through the blood. They then attach to the receptors in the brain, leading to addiction and abuse.
The company said the drug, named VX-548, was safe and well-tolerated in trials that tested a total of about 2,400 patients. Common side effects included nausea and constipation.
Attempts to bring new opioid alternatives to market by drugmakers such as Eli Lilly (NYSE:LLY) and Regeneron (NASDAQ:REGN) Pharmaceuticals have failed in trials.
"I don't think anybody expects that this drug will replace or mean the end of opioid pain medicines, but it absolutely offers an alternative that is sorely needed," Piper Sandler analyst Christopher Raymond said ahead of data.
Roughly 80 million patients are prescribed a medicine for their moderate-to-severe acute pain every year in the U.S, Vertex said.