SPG601 granted orphan drug status in EU for treating fragile X
Designation follows positive top-line findings from small clinical trial
SPG601, an oral treatment candidate now in clinical testing, has been granted orphan drug status for fragile X syndrome in the European Union.
This designation, awarded by the European Medicines Agency (EMA), is meant to incentivize companies developing treatments for rare diseases — those affecting fewer than 5 of every 10,000 people in the EU — and that lack effective therapies.
Orphan drug status comes with several benefits, including regulatory fee reductions and a guarantee that, if SPG601 is approved in the EU, its developer Spinogenix would be entitled to a decade of market exclusivity.
“We are grateful to the EMA for recognizing the importance of developing treatments for [fragile X] and supporting the development and path to market for our novel drug, SPG601,” Stella Sarraf, PhD, founder and CEO of Spinogenix, said in a company press release.
SPG601 was previously granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA also awarded the therapy fast track status in the U.S., which aims to speed the development of new treatments that can potentially improve care for serious conditions.
“We want to ensure that every person living with this disabling condition can access treatment,” Sarraf said. “Having already obtained [orphan drug] and fast track designations from the U.S. FDA, this new grant from the EMA takes us one step forward to achieving our mission to help patients across the globe.”
Trial involving 10 patients evaluated safety, efficacy of SPG601
The new designation comes just a few months after Spinogenix announced positive top-line findings from a Phase 2a clinical trial (NCT06413537) assessing the safety and efficacy of SPG601 in people with fragile X.
That study, conducted in the U.S. in Ohio, enrolled 10 men with fragile X syndrome, ages 18-45. Each was randomly assigned to take one dose of SPG601 (800 mg, taken as eight capsules) or a placebo. A week later, participants given SPG601 were given the placebo and vice versa. Electrical activity in the patients’ brains was recorded using an electroencephalogram, or EEG.
The results showed that SPG601 was able to reduce high frequency gamma band activity — an abnormal pattern of electrical activity in the brain that is associated with learning and memory problems in fragile X.
Gamma band activity is thought to occur in fragile X syndrome because nearby nerve cells form too many immature connections with each other. This occurs at the expense of mature connections established between nerve cells that are farther away from each other.
Currently, [fragile X syndrome] has no approved treatment, making indications like the [European Medicines Agency’s orphan drug designation] crucial for advancing therapies and accelerating development.
The experimental therapy is designed to help normalize the formation of connections between nerve cells, which are known as synapses. It does so by increasing the activity of large-conductance calcium-activated potassium channels, or BK channels, which are key proteins involved in synaptic function.
According to Craig Erickson, MD, chief medical advisor at Spinogenix and the trial’s principal investigator, “SPG601 has the potential to improve the underlying synaptic deficits central to this condition, offering immense hope to the whole patient community.”
Erickson presented the study’s findings at the Cincinnati Fragile X Family Conference, held earlier this month at Cincinnati Children’s Hospital.
“Currently, [fragile X syndrome] has no approved treatment, making indications like the EMA’s [orphan drug designation] crucial for advancing therapies and accelerating development,” Erickson said.
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