US Food and Drug Administration granted Breakthrough Therapy Designation(BTD) for VER-01 (the flagship full-spectrum marijuana drug) from Munich-based Vertanical. This puts it in the prime position of becoming the FDA’s first approved cannabis-derived medication for pain and only the 2nd such approval after Epidiolex, in 2018.
The FDA designation for VER-01 was granted to the investigational medicine that shows a substantial improvement in treatment over current therapies for serious diseases on 18th May 2026.
Vertanical is not yet FDA-approved for its treatment of chronic low back discomfort that does not use opioids. However, this marks an important milestone for the company as it prepares to receive marketing authorization in Europe within weeks.
It is just a few weeks since the US moved medical marijuana to Schedule III and recognized cannabis’ medical benefits.
You can also read about the advantages of using MEDCAN24 VER-01, as reported in December 2012, is the result of two Phase III randomised controlled studies (RCTs).
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The results of a controlled study, which included 820 patients and was published in Nature Medicine In September 2025, VER-01 achieved its primary endpoint. Its effects lasted for a long time and showed significant improvement in physical and sleep function.
A second study comparing VER-01 to opioids in 384 patients from Germany, Poland, Czech Republic and Spain found VER-01 provided superior pain relief, better gastrointestinal tolerateability and no sign of addiction or withdrawal.
Vertanical’s Founder and Chief Executive of FUTRUE, Dr Clemens Fisher said: “We think VER-01 can change the way chronic pain is managed and provide physicians with a non-opioid alternative.”
Vertanical, a company in the United States, is also preparing to launch its groundbreaking treatment on European markets. The firm says that this will happen within weeks.
The EU Mutual Recognition Procedure provides a route for broader access to the continental market once Germany approves the product as a medicinal product. VER-01, which will be marketed as Exilby and cost more than $250m to develop over seven years, is now ready for the market.
Vertanical’s chronic low-back pain treatment is aimed at a much larger market than Epidiolex. Jazz Pharmaceuticals, the market leader in CBD treatments for rare epilepsies and generating sales of approximately $100 million per year, has a CBD product that leads on this market.
Vertanical, in the US, has begun an additional pivotal phase 3 trial, to meet FDA requirements. The first readout of data is expected to be 2027, and if positive, an NDA will follow 2028.
In the United States, more than 60 millions people suffer chronic pain. Yet one fifth of patients who experience acute pain still receive opioids as a treatment for lack of alternative options.
BTD has a particular implication, which is not well-covered in the broader rescheduling discussion. Speakers with regulatory experience made a crucial distinction at Cannabis Europa London in London, this week.
Saphira Galoob (Executive Director of US Cannabis Roundtable) explained at the event that the order in April covers two categories: FDA-approved cannabis-containing drugs and products licensed under state-licensed market.
Jonathan Havens who founded the cannabis practice at Saul Ewing, and was regulatory counsel with the FDA, before going into private practice, said that the FDA had only ever approved Epidiolex.
Synthetic analogs, such as Marinol, and Syndros have also been placed on Schedule III. However, they are not made from the cannabis plants.
VER-01, through its NDA, can be placed in the first category, if it is accelerated with BTD. FDA-approved Schedule III medications qualify for hospital formularies and insurance reimbursement negotiation, as well as prescribing from any licensed physician.
Rescheduling opened up a new commercial aspect, but it is still incomplete. Galoob pointed out that medical cannabis can be exported under US law.
Havens noted also that the DEA final order contemplates the import and export of registered producers, exporters and importers. However, the registration portal was only recently opened and the initial wave only covered dispensaries.
Havens, a panelist at a packed discussion on the subject of national security and international affairs told attendees that “the final order states that this is contemplated. That’s technically allowed today.”
I don’t believe there is an easy way until we hear more from the DEA about who they are deciding to register.
Vertanical, however, has a BTD designation that offers it more protection against the constantly changing politics surrounding cannabis re-scheduling.
VER-01, a full spectrum extract standardised 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. Orally, it is taken as drops of liquid. Vertanical makes it clear that the findings of its study cannot be applied to other cannabis products or flower extracts.
The next important milestone will be European marketing authorization. The mutual recognition dossier, if Germany moves as quickly as predicted in the coming weeks, will decide how fast the rest the EU will move.