Ionis treatment for Angelman syndrome receives orphan drug and rare pediatric disease designations from U.S. FDA
Angelman syndrome, which affects an estimated one in 12,000 to 20,000 people globally,1 presents early in life with profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. It is a neuro-developmental disorder that generally does not improve following symptom onset in early childhood, resulting in complete dependence on a caregiver. Currently, there are no disease-modifying treatments for Angelman syndrome.
"Receiving FDA orphan drug designation for ION582 reflects the important and urgent need for delivering an effective treatment to patients living with Angelman syndrome. Ionis is committed to working closely with regulators, clinical investigators, patients and caregivers to advance this novel treatment and make it available to those who need it," said
ION582 is being evaluated in a Phase 1/2, open-label, dose-escalation clinical study in up to approximately 44 participants with Angelman syndrome. For more information on the HALOS Study (NCT05127226), visit clinicaltrials.gov.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming a leading, fully integrated biotechnology company.
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1 Mertz LG, Christensen R, Vogel I, Hertz JM, Nielsen KB, Gronskov K, Ostergaard JR. Angelman syndrome in
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