FDA Grants Rare Pediatric Disease Designation to Ionis Treatment for Angelman Syndrome

The U.S. Food and Drug Administration granted Orphan Drug and Rare Pediatric Disease designations to Ionis Pharmaceuticals’ investigational antisense medicine ION582 for the treatment of Angelman syndrome, a rare neurogenetic disorder caused by the loss of function of the maternally inherited ubiquitin protein ligase E3A gene.

Photo: C. Frank Bennett, executive vice president, chief scientific officer, and franchise leader for neurological programs at Ionis Pharmaceuticals

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, executive vice president, chief scientific officer, and franchise leader for neurological programs at Ionis.

ION582, which targets UBE3A, is being evaluated in a phase 1/2, open-label, dose-escalation clinical study in up to approximately 44 participants with Angelman syndrome.

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 pediatric disease as a serious or life-threatening disease that primarily affects individuals from birth to 18 years of age.

Under the FDA’s rare pediatric 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. Most recently, Bridgebio Pharma sold its priority review voucher for $110 million.

Author: Rare Daily Staff