US FDA grants orphan drug and rare paediatric disease designations to Ionis Pharma ION582 to treat Angelman syndrome

Ionis Pharmaceuticals, Inc., the leader in RNA-targeted therapeutics, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation and rare paediatric disease designation to ION582, an investigational antisense medicine for the treatment of Angelman syndrome, a rare neurogenetic disorder caused by the loss of function of the maternally inherited Ubiquitin Protein Ligase E3A (UBE3A) gene. Angelman syndrome, which affects an estimated one in 12,000 to 20,000 people globally, 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 C. Frank Bennett, Ph.D., executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis. Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval. The FDA defines a rare paediatric disease as a serious or life-threatening disease that primarily affects individuals from birth to 18 years of age. Under the FDA's rare paediatric disease designation and voucher programs, if Ionis receives marketing approval for ION582 for Angelman syndrome, the company may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. 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 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.

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