GRÄFELFING, Germany — The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to VER-01, Vertanical’s non-opioid, cannabinoid-extract-based investigational treatment for chronic low back pain.
The designation follows two randomized, controlled Phase 3 trials demonstrated significant pain reduction, a favorable tolerability profile, and no evidence of dependence. In a direct Phase 3 head-to-head comparator study, VER-01 also showed superior pain reduction and better gastrointestinal tolerability than opioids, reinforcing its potential as a differentiated non-opioid alternative in chronic pain care.
The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of investigational medicines where preliminary clinical evidence indicates the potential for substantial improvement over available therapies.
Chronic pain remains one of the largest unmet needs in healthcare. More than one billion people worldwide live with chronic pain, including more than 60 million Americans. For many patients, opioids remain central in pain management despite well-documented risks, including dependence, abuse potential, and debilitating side effects.
The need for effective, well-tolerated non-opioid treatment options remains especially urgent in the United States. Despite its enormous medical, social, and economic burden, innovation in chronic pain treatment has substantially lagged behind other major disease areas.
In a placebo-controlled European Phase 3 study published last September in Nature Medicine, VER-01 demonstrated significant reductions in pain compared with placebo and met the study’s primary endpoint, with effects sustained during long-term treatment. Patients receiving VER-01 also experienced significant improvements in key symptoms associated with chronic pain, including sleep disturbances and impaired physical function.
In a second Phase 3 head-to-head comparator study, VER-01 demonstrated greater pain reduction and better gastrointestinal tolerability compared with opioids. Across the clinical program, VER-01 was generally well tolerated, with no evidence of dependence or withdrawal symptoms observed.
As Vertanical expects marketing authorization in the first European countries in the coming weeks, the company has also initiated an additional pivotal Phase 3 trial in the United States to support a future FDA submission. The study is designed to confirm the efficacy and safety of VER-01 in U.S. patients with chronic low back pain to address U.S. regulatory requirements.
Based on the current development plan, the company anticipates a first data read-out in 2027 and, subject to positive results, plans to submit a New Drug Application in 2028.
About VER-01’s mechanism
VER-01 is an investigational standardized full-spectrum extract derived from the Cannabis sativa strain DKJ127 L., a unique and proprietary plant genetics selected to provide a phytochemical profile tailored to chronic pain. Its composition includes a defined mixture of cannabinoids, terpenes and other bioactive compounds selected for their potential relevance in pain modulation and associated symptoms. VER-01 has been comprehensively characterized using chromatographic and spectrometric methods to quantify key constituents and ensure pharmaceutical-grade quality and product consistency. This level of standardization is critical, as cannabis-derived products differ substantially in their pharmacological profile, composition of bioactive constituents and product quality, depending on plant genetics, raw materials and manufacturing processes. Findings obtained with VER-01 can therefore not be extrapolated to other cannabis extracts or products.
