EU Orphan Drug Status Sought for Nova Therapy Using Hallucinogenic Psilocybin

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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psilocybin therapy/ seeking EU orphan designation for hallucinogenic compound

Nova Mentis is seeking orphan drug designation from the European Medicines Agency (EMA) for psilocybin, its investigational therapy for fragile X syndrome.

Psilocybin, found naturally in certain mushrooms — popularly called “magic mushrooms” — is most known for its hallucinogenic, anxiolytic, and psychoactive effects. However, Nova officials said in a press release announcing the filing that their compound has been shown to be effective “without apparent psychedelic side effects.”

The biotech company recently filed its application for orphan status with the EMA, which is responsible for the scientific evaluation of all medicines in the European Union.

Orphan drug designation would give Nova incentives to further develop psilocybin, including “assistance with trial protocols, reduced regulatory fees and 10 years of market exclusivity after drug approval,” said Will Rascan, CEO and president.

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Fragile X is the most common cause of inherited intellectual disability and autism spectrum disorder (ASD). While there currently is no cure for fragile X, psychiatric approaches to ASD have been found to ease some symptoms. Other approaches include sensory integration training and special education, occupational, speech, and behavior therapy.

Now, Nova’s research and drug development program is exploring “the clinical potential of psilocybin for chronic neuroinflammatory conditions with unmet medical needs,” Rascan said.

According to the company, psilocybin may be a new, first-in-class treatment for fragile X.

While psilocybin is well known as a hallucinogen, its use in a preclinical model of fragile X “has revealed positive improvement in cognition without apparent psychedelic side effects,” said Marvin S. Hausman, MD, chairman of Nova’s scientific advisory board.

Besides the potential benefits for cognition, psilocybin also appears to be able to modulate inflammation and the gut microbiota — the community of microbes living in the gut. The gut microbiota is thought to communicate with the brain via the microbiota-gut-brain axis, and many people with fragile X have gastrointestinal disorders.

“I am also excited to report that psilocybin-treated animals showed changes in inflammatory biomarkers that are potentially involved in the ASD disease process,” Hausman said.

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Psilocybin is thought to increase brain plasticity and reduce anxiety and behavioral rigidity. Of note, synaptic plasticity refers to the ability of synapses — the junctions between two nerve cells that allow them to communicate — to strengthen or weaken over time.

Nova calls it “a significant step above currently available methodologies.”

To aid the development of patient-tailored treatments, Nova Mentis also is creating a data bank of biomarkers associated with fragile X and ASD.

In the near future, the company plans to submit regulatory paperwork supporting the potential efficacy of psilocybin both to EMA and the U.S. Food and Drug Administration (FDA), and to start clinical trials in patients with fragile X and ASD.