Spinogenix announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to SPG601 for the treatment of people with Fragile X syndrome (FXS), a condition for which there is currently no approved medicine.
FXS, a known cause of autism, is the leading inherited form of intellectual disability caused by the silencing of the Fmr1 gene. FXS can produce a wide range of disabling symptoms, with many individuals requiring lifelong around-the-clock supportive care.
SPG601 works at the synaptic level, targeting a well-established molecular dysfunction in FXS, addressing core symptoms to improve challenging behaviors and the overall quality of life of those affected. It is a small molecule large-conductance, calcium-activated potassium ("BK") channel activator that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions that underlie many core symptoms of FXS.
"We are grateful to the EMA for recognizing the importance of developing treatments for FXS and supporting the development and path to market for our novel drug, SPG601," said Dr. Stella Sarraf, Spinogenix Chief Executive Officer and Founder. "We want to ensure that every person living with this disabling condition can access treatment. Having already obtained ODD 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."
Topline results from a Phase 2 randomized, double-blind, placebo-controlled, crossover study of SPG601 therapy in adult men with FXS were announced earlier this year. The study met its primary goal, significantly reducing high-frequency gamma band activity in the FXS subjects, a key indicator that occurs in FXS patients at the expense of normal brain activity levels that are used for learning and memory.
"Currently, FXS has no approved treatment, making indications like the EMA's ODD crucial for advancing therapies and accelerating development," said Dr. Craig Erickson, Spinogenix Chief Medical Advisor and principal investigator of the recently completed Phase 2 study conducted at Cincinnati Children's Hospital. "SPG601 has the potential to improve the underlying synaptic deficits central to this condition, offering immense hope to the whole patient community."
The EMA grants ODD to drugs and biologics intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that affects fewer than five in 10,000 people in the European Union, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. Companies that secure ODD benefit from a 10-year period of market exclusivity following authorization, protocol assistance, and regulatory fee reductions.
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