FDA Grants Breakthrough Therapy Designation to VERTANICAL's VER-01, a First-in-Class Non-Opioid Investigational Treatment for Chronic Low Back Pain
GRÄFELFING, Germany, May 18, 2026 /PRNewswire/ -- VERTANICAL today announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to VER-01, the company's first-in-class non-opioid investigational treatment for chronic low back pain.
The FDA Breakthrough Therapy Designation has been granted after 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. For VERTANICAL, the designation represents an important regulatory milestone in its U.S. development program and underscores the potential of VER-01 in a therapeutic area where meaningful innovation has remained limited for decades.
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. With its novel mechanism of action, VER-01 represents a differentiated investigational approach in a field still shaped by long-standing treatment paradigms.
"The FDA's Breakthrough Therapy Designation for VER-01 is a major recognition of its potential to address the significant unmet need in chronic pain," said Dr. Clemens Fischer, CEO of FUTRUE Group and Founder of VERTANICAL. "Patients have waited far too long for meaningful progress. We believe VER-01 has the potential to change how chronic pain is treated and offer physicians a much-needed non-opioid solution."
The designation is supported by results from VERTANICAL's European Phase 3 clinical program in chronic low back pain. In a placebo-controlled 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.
The pivotal U.S. Phase 3 trial represents the next major step in VERTANICAL's U.S. development program. 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.
The FDA Breakthrough Therapy Designation represents an important recognition of VER-01's potential and marks a key step in VERTANICAL's efforts to advance a first-in-class non-opioid treatment option for U.S. patients, in a market where the need for effective and well-tolerated alternatives to opioids remains particularly urgent.
About VER-01
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.
About VERTANICAL
VERTANICAL is a leading biopharmaceutical company dedicated to developing and commercializing innovative therapies for chronic pain. Guided by the vision of a world free of chronic pain, the company is committed to advancing clinical research and development in chronic pain diseases. With over 20 completed preclinical and clinical studies and a comprehensive development program across multiple chronic pain indications, VERTANICAL is recognized as one of the global innovation leaders in the field of chronic pain.
VERTANICAL was founded in 2007 by Dr. Clemens Fischer and has its global headquarters in Munich, Germany. The company operates one of the world's most advanced GMP facilities for the development and manufacturing of biopharmaceuticals. It maintains an international network of several hundred research and clinical trial sites across the U.S. and multiple European countries.
Media contact: Vertanical GmbH Am Haag 14 82166 Gräfelfing Germany
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