The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to VER-01, the flagship full-spectrum cannabis drug from Munich-based Vertanical, putting it in prime position to become the first FDA-approved cannabis-derived medicine for pain, and only the second such approval since Epidiolex in 2018.
VER-01’s FDA designation, granted on 18 May 2026, is reserved for investigational medicines showing the potential for substantial improvement over existing therapies in serious conditions.
While this does not mean Vertanical has now achieved FDA approval for its non-opioid treatment for chronic low back pain, it marks a significant milestone as the company prepares for marketing authorisation in its first European markets within weeks.
The timing comes just weeks after the US officially moved medical cannabis to Schedule III, recognising cannabis treatment’s medical benefits for the first time in its history.
VER-01’s designation follows two Phase 3 randomised controlled trials (RCTs).
In a placebo-controlled study of 820 patients published in Nature Medicine in September 2025, VER-01 met its primary endpoint with effects sustained over the long term and significant improvements in sleep and physical function.
A second head-to-head comparator study, the ELEVATE trial, across 384 patients in Germany, the Czech Republic, Poland and Spain, found VER-01 delivered superior pain reduction and better gastrointestinal tolerability compared with opioids, with no evidence of dependence or withdrawal.
“We believe VER-01 has the potential to change how chronic pain is treated and offer physicians a much-needed non-opioid solution,” said Dr Clemens Fischer, Chief Executive Officer of FUTRUE Group and Founder of Vertanical.
Across the Atlantic, Vertanical is also on the verge of launching its breakthrough treatment in European markets, which the company says is expected within weeks.
Once Germany grants approval as an authorised medicinal product, the EU mutual recognition procedure provides the route to broader continental access. VER-01 will be marketed under the name Exilby, and has been seven years in development at a cost exceeding $250m
With Epidiolex, Jazz Pharmaceuticals’ market-leading CBD treatment for rare epilepsies, generating approximately $100m in annual sales, Vertanical is targeting a significantly larger market with a chronic low back pain treatment, which affects an estimated half a billion people worldwide.
In the US, Vertanical has initiated an additional pivotal Phase 3 trial to satisfy FDA requirements, with a first data read-out expected in 2027 and an NDA planned for 2028, subject to positive results.
More than 60 million Americans suffer from chronic pain, one in five patients with acute pain are still prescribed opioids for want of alternatives.
The BTD also carries a specific implication that has received little attention in the wider rescheduling coverage.
At Cannabis Europa London last week, speakers with direct regulatory experience drew a distinction that matters for any European company watching the US market develop.
Speaking at the event, Saphira Galoob, Executive Director of the US Cannabis Roundtable, explained that the April order covers two categories, FDA-approved drugs containing cannabis and products regulated under state-licensed markets.
“There’s really only one true cannabis-derived drug product that FDA has ever approved, which is Epidiolex,” said Jonathan Havens, who co-founded the cannabis law practice at Saul Ewing and served as regulatory counsel at the FDA before entering private practice.
Synthetic analogues such as Marinol and Syndros are also already Schedule III, he noted, but are not derived from the cannabis plant.
VER-01’s NDA pathway, accelerated by the BTD, is a route to joining Epidiolex in that first category. FDA-approved Schedule III medicines qualify for standard hospital formularies, insurance reimbursement negotiations, and prescribing by any licensed physician, without the compliance architecture that state-licensed operators must navigate.
There is a further commercial dimension that rescheduling has opened, though it remains incomplete in practice. Galoob noted that under US law, medical cannabis products can now be exported.
Havens also noted that the DEA’s final order contemplates import and export for registered producers, importers and exporters, but the registration portal has only just opened, and the first wave covered dispensaries only.
“The final order says that it’s contemplated, that’s allowed technically today,” Havens told delegates during a packed panel discussion.
“I don’t think there’s an easy way to do it until we start to hear from the DEA on who they’re deciding to register.”
However, Vertanical’s BTD designation offers its route to market more protection from the ever-shifting politics of cannabis rescheduling.
VER-01 is a standardised full-spectrum extract from the proprietary Cannabis sativa strain DKJ127 L., containing a defined mixture of cannabinoids, terpenes and bioactive compounds, including cannabigerol, β-caryophyllene and α-bisabolol. It is administered orally as liquid drops. Vertanical is explicit that its findings cannot be extrapolated to other cannabis extracts or prescribed flower products.
European marketing authorisation will be the next significant milestone to watch. Should Germany proceed as expected in the coming weeks, the mutual recognition dossier will determine how quickly the rest of the EU follows.
