Breakthrough Therapy Designation Granted to Non-opioid Treatment for Chronic Low Back Pain
KEY TAKEAWAYS
- The FDA granted Breakthrough Therapy designation to VER-01, an investigational non-opioid therapy for chronic low back pain.
- Phase 3 studies showed significant pain reduction and favorable gastrointestinal tolerability compared with opioids.
- No evidence of dependence or withdrawal symptoms was observed across the clinical program.
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to VER-01 (Vertanical, Munich, Germany), an investigational non-opioid medication for chronic low back pain. According to the company, the designation was supported by findings from 2 randomized phase 3 trials demonstrating significant pain reduction, favorable tolerability, and no evidence of dependence.
VER-01 is a pharmaceutical-grade, standardized extract derived from a proprietary Cannabis sativa strain. According to Vertanical, the product is manufactured to maintain a consistent profile of cannabinoids, terpenes, and other bioactive compounds, and findings from its clinical program should not be generalized to nonstandardized cannabis products or extracts.
Findings from the clinical program
- In a placebo-controlled phase 3 trial (NCT04940741) published in Nature Medicine, VER-01 met its primary end point, with greater pain reduction than placebo in people with chronic low back pain. The company also reported maintained benefit during longer-term treatment.
- Participants treated with VER-01 also had reported improvements in pain-related sleep problems and physical function.
- In a separate phase 3 comparator trial (NCT05610813), VER-01 was reported to outperform opioid therapy on pain reduction and to have fewer gastrointestinal tolerability concerns.
- Across the company’s clinical program, Vertanical reported that VER-01 was generally well tolerated and that no dependence or withdrawal signal was observed.
The company stated that an additional pivotal US phase 3 study is underway, with data expected in 2027 and a potential New Drug Application submission planned for 2028.
Source
Vertanical. FDA grants breakthrough therapy designation to Vertanical’s VER-01, a first-in-class non-opioid investigational treatment for chronic low back pain. PR Newswire. Published May 18, 2026. Accessed May 18, 2026. https://www.prnewswire.com/news-releases/fda-grants-breakthrough-therapy-designation-to-vertanicals-ver-01-a-first-in-class-non-opioid-investigational-treatment-for-chronic-low-back-pain-302774621.html