Ionis Pharmaceuticals announced that its investigational medicine, ION582, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome. This designation is intended to accelerate the development and review of new drugs and biologics that treat serious conditions and fill an unmet medical need.
Mechanism of Action
ION582 is an antisense oligonucleotide (ASO) designed to reduce the expression of the UBE3A-AS transcript in individuals with Angelman syndrome. Angelman syndrome is a rare neurogenetic disorder caused by loss-of-function of the maternally inherited UBE3A gene. Suppressing the UBE3A-AS transcript aims to unsilence the normally silenced paternal UBE3A allele, thus restoring UBE3A protein levels in the brain.
Clinical Significance
Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 live births. Characteristics of Angelman syndrome include developmental delay, intellectual disability, severe speech impairment, movement disorders, and seizures. Currently, there are no approved therapies that address the underlying genetic cause of Angelman syndrome, highlighting the significant unmet medical need.
The Fast Track designation by the FDA is a crucial step in expediting the development of ION582, potentially bringing a much-needed therapy to patients and families affected by Angelman syndrome sooner. Ionis Pharmaceuticals is actively advancing the clinical development program for ION582, with ongoing trials assessing its safety and efficacy.
