FDA Grants Breakthrough Therapy Designation to Investigational Cannabis-Derived Therapy for Chronic Low Back Pain

The FDA has granted Breakthrough Therapy Designation (BTD) to VER-01, an investigational cannabis-derived treatment for chronic low back pain (CLBP), following positive findings from 2 phase 3 clinical trials, a placebo-controlled phase 3 trial (NCT04940741) and the ELEVATE trial (NCT05610813). The BTD is intended to expedite development and review of therapies that may offer substantial improvement over available treatments for serious conditions.¹

If VER-01 is ultimately approved, it could become the first FDA-approved cannabis-derived therapy specifically indicated for pain management in the United States, which would represent a novel nonopioid option for patients with CLBP, a condition affecting more than 500 million individuals worldwide.^2,3

The designation arrives amid growing interest in nonopioid pain therapies as clinicians seek alternatives to opioid analgesics, which remain associated with risks of misuse, dependence, overdose, and adverse effects.⁴

What Is VER-01?

VER-01 is a standardized, full-spectrum extract derived from a proprietary strain of Cannabis sativa known as DKJ127 L. The investigational therapy contains a defined mixture of cannabinoids, terpenes, and other bioactive compounds, including cannabigerol (CBG), β-caryophyllene, and α-bisabolol, unlike individual cannabinoid products.¹

The therapy is administered orally as liquid drops and has been developed under pharmaceutical manufacturing standards to ensure consistency between batches. According to the manufacturer, the clinical findings observed with VER-01 should not be extrapolated to other cannabis extracts, medical marijuana products, or cannabis flower formulations.¹

Acknowledging this distinction is vital for health specialists because variability in cannabinoid content and formulation has historically complicated the interpretation of cannabis-related clinical data. VER-01 represents a standardized pharmaceutical product evaluated through the traditional FDA drug development pathway.

Phase 3 Clinical Trial Findings

The support for the FDA designation stems from the ELEVATE phase 3 trial and a placebo-controlled phase 3 trial which evaluated VER-01 in patients with CLBP.¹

The first data set from the randomized, double-blind, placebo-controlled study published in Nature Medicine had enrolled 820 adults with CLBP and compared VER-01 with placebo. The data reported that the trial met its primary efficacy end point, demonstrating statistically significant reductions in pain intensity when compared with placebo after 12 weeks of treatment. Benefits were sustained during long-term follow-up, and researchers had also observed improvements in sleep quality and physical function.²

Additionally, the data provided no evidence of dependency or withdrawal associated with treatment. While adverse effects occurred more frequently among patients receiving VER-01 than the placebo, most were mild to moderate and transient.²

The second phase 3 study evaluated VER-01 against opioid therapy in 384 patients across multiple European countries. Researchers reported superior pain reduction with VER-01 compared with opioids, along with improved gastrointestinal tolerability and better sleep outcomes. The data also found no evidence of dependence or withdrawal.⁵

When taken together, the findings from this data indicate VER-01 may address several limitations associated with current pharmacologic treatment options for chronic pain.

Why This Matters

Should VER-01 eventually receive FDA approval, health care practitioners may encounter questions in regard to its mechanism, safety profile, formulary positioning, and potential role relative to existing analgesics.

The BTD does not constitute FDA approval, and additional clinical development remains underway. Vertanical has initiated another pivotal phase 3 trial in the United States, with data expected in 2027 and a potential New Drug Application submission planned for 2028, contingent on positive results.¹

Nevertheless, the designation signals growing regulatory recognition of cannabis-derived pharmaceuticals developed through rigorous clinical research. For pharmacists, VER-01 represents an emerging example of how cannabinoid-based therapies may evolve beyond state-regulated cannabis programs into evidence-based prescription medicines evaluated under FDA standards.

REFERENCES

1. 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. May 18, 2026. Accessed June 8, 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

2. Karst M, Meissner W, Sator S, et al. Full-spectrum extract from Cannabis sativa DKJ127 for chronic low back pain: a phase 3 randomized placebo-controlled trial. Nat Med. 2025;31:4189–4196. doi:10.1038/s41591-025-03977-0

3. World Health Organization. Low back pain fact sheet. Accessed June 8, 2026. https://www.who.int/news-room/fact-sheets/detail/low-back-pain

4. Dowell D, Ragan KR, Jones CM, Baldwin GT, Chou R. CDC Clinical Practice Guideline for Prescribing Opioids for Pain — United States, 2022. MMWR Recomm Rep 2022;71(No. RR-3):1–95. doi:10.15585/mmwr.rr7103a1

5. Meissner W, Argoff C, Sator S, Schoder V, Karst M. VER-01 Shows Enhanced Gastrointestinal Tolerability, Superior Pain Relief, and Improved Sleep Quality Compared to Opioids in Treating Chronic Low Back Pain: A Randomized Phase 3 Clinical Trial. Pain Ther. 2025;14(6):1765-1782. doi:10.1007/s40122-025-00773-z