The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to VER-01, an investigational cannabis-based chronic pain treatment developed by VERTANICAL. This designation applies to VER-01 as a non-opioid treatment for chronic low back pain. The decision follows the presentation of data from two randomized, controlled Phase 3 trials.
FDA Breakthrough Therapy Designation for VER-01
The FDA’s Breakthrough Therapy Designation is intended to accelerate the development and review process for investigational medicines that show preliminary clinical evidence of offering substantial improvement over existing therapies for serious conditions. For VERTANICAL, this designation indicates the FDA’s recognition of VER-01’s potential in an area with significant unmet medical need.
- Chronic pain affects over one billion people globally, including more than 60 million Americans.
- Opioids remain a primary treatment option for many patients, despite documented risks such as dependence and side effects.
- Innovation in chronic pain treatment has lagged compared to other major disease areas, highlighting the demand for effective non-opioid alternatives.
Dr. Clemens Fischer, CEO of FUTRUE Group and Founder of VERTANICAL, stated that the designation is a recognition of VER-01’s potential to address the unmet need in chronic pain management.
Clinical Evidence Supporting the Cannabis-Based Chronic Pain Treatment
The Breakthrough Therapy Designation is supported by results from VERTANICAL’s European Phase 3 clinical program. These trials evaluated VER-01, a standardized full-spectrum extract derived from the Cannabis sativa strain DKJ127 L., which contains a defined mixture of cannabinoids, terpenes, and other bioactive compounds.
Phase 3 Trial Outcomes
A placebo-controlled Phase 3 study, published in Nature Medicine last September, demonstrated that VER-01 provided significant reductions in pain compared to placebo. The study met its primary endpoint, with effects maintained during long-term treatment. Patients receiving VER-01 also experienced improvements in associated symptoms, including sleep disturbances and physical function.
In a second Phase 3 head-to-head comparator study, VER-01 showed greater pain reduction and improved gastrointestinal tolerability when compared to opioids. Across the clinical program, VER-01 was generally well tolerated, with no evidence of dependence or withdrawal symptoms observed.
U.S. Development Pathway and Future Outlook
VERTANICAL has initiated an additional pivotal Phase 3 trial in the United States. This study is designed to confirm the efficacy and safety of VER-01 in U.S. patients with chronic low back pain, addressing specific U.S. regulatory requirements.
The company anticipates a first data read-out from this U.S. trial in 2027. Subject to positive results, VERTANICAL plans to submit a New Drug Application (NDA) to the FDA in 2028. This timeline reflects the expedited development pathway afforded by the Breakthrough Therapy Designation, which aims to facilitate the availability of non-opioid options for chronic pain.
VERTANICAL anticipates receiving marketing authorization for VER-01 in initial European countries in the coming weeks.
Disclaimer: This article is for informational purposes only and does not constitute medical advice. Hemp Gazette does not provide medical recommendations, diagnoses, or treatment plans. Always consult a qualified healthcare practitioner before making any decisions regarding your health or any medical condition. Statements concerning the therapeutic uses of hemp, cannabis, or cannabinoid-derived products have not been evaluated by Australia’s Therapeutic Goods Administration (TGA). Medicinal cannabis products in Australia are accessed via prescription pathways under TGA regulation.
