FDA grants «breakthrough therapy» status to cannabis-based pain treatment VER-01

The Food and Drug Administration (FDA) American granted the designation of «innovative therapy» at VER-01, a cannabis-derived treatment developed by the German pharmaceutical company Vertanical for chronic back pain.

The FDA's Breakthrough Therapy designation is reserved for investigational drugs intended to treat serious conditions, and which have shown substantial improvement over existing treatments in preliminary clinical data. While this status does not constitute marketing authorization, it does enable closer collaboration with regulatory authorities and an accelerated review process once final trial results are available.

For Vertanical, this decision strengthens the company's position as it prepares for both a European launch and the continuation of its clinical development program in the United States. There, VER-01 could become the first cannabis-based treatment approved specifically for pain management.

VER-01 shows promising results against chronic pain

The VER-01, which we have already mentioned here,is a standardized, full-spectrum extract derived from Jack Herer variety (patented variety DKJ127). The sesame oil-based oral formulation contains 5% of THC, and each dose delivers 2.5 milligrams of THC, 0.1 mg of cannabigerol and 0.02 mg of cannabidiol. It is accompanied by the flower's natural terpenes, notably β-caryophyllene and α-bisabolol.

The FDA based its decision on the results of two Phase 3 clinical trials. The first, a placebo-controlled study involving 820 patients and published in Nature Medicine in 2025, showed a significant reduction in chronic low-back pain. Researchers also observed improvements in sleep quality and physical function, two symptoms commonly associated with chronic pain conditions.

A second Phase 3 study, called ELEVATE, compared the VER-01 directly to opioid-based treatment in 384 patients in Germany, Poland, Spain and the Czech Republic. According to Vertanical, the cannabis-derived drug delivered greater pain reduction and better gastrointestinal tolerance than opioids.

The clinical program also reported no signs of dependence, withdrawal symptoms or risk of abuse, an important aspect as healthcare systems continue to look for ways to reduce the risk of addiction. alternatives to opioid drugs.

«We believe VER-01 has the potential to change the way chronic pain is treated and offer physicians a much-needed non-opioid solution,» said Dr Clemens Fischer, CEO of the FUTRUE Group and founder of Vertanical.

European launch imminent under the Exilby

The FDA announcement comes as Vertanical prepares to launch the product in Europe under the trade name Exilby. The Munich-based company is said to have invested over $250 million and devoted seven years of research to the program.

Germany is expected to be the first market where Exilby will obtain approval as a finished pharmaceutical product. In the event of approval, Vertanical plans to use the European Union's mutual recognition procedure to extend access to other member states.

Unlike prescription cannabis flowers, Exilby would be marketed as a licensed pharmaceutical drug, placing it within a more conventional regulatory framework for doctors, pharmacists and health insurers.

A growing market beyond’Epidiolex

If it is finally approved by the FDA, the VER-01 would become the second cannabis-derived medicine to be approved in the United States after’Epidiolex, CBD-based treatment for epilepsy approved in 2018.

The business opportunity could be considerable. Chronic low back pain affects around 500 million people worldwide, and remains one of the leading causes of disability. In the USA alone, over 60 million people suffer from chronic pain, while opioid prescriptions continue to play a major role in pain management despite long-standing concerns about the risks of addiction and overdose.

Vertanical has already launched an additional phase 3 study required by the FDA. The company expects to obtain preliminary data in 2027 and, if the results remain positive, plans to file a marketing authorization application in 2028.