- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) share price rose more than 3.5% before trading today. This is why.
The share price of Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a leader in RNA-targeted therapeutics, announced today that the US Food and Drug Administration (FDA) increased more than 3.5% before of marketing today. Investors are responding positively to Ionis Pharmaceuticals granting Orphan Drug Designation and Rare Pediatric Disease Designation to ION582, an investigational antisense drug for the treatment of Angelman syndrome, a rare neurogenetic disorder caused by loss of function of the maternally inherited ubiquitin protein ligase E3A (UBE3A). ) gene. ION582 points to UBE3A.
Affecting approximately one in 12,000 to 20,000 people worldwide, Angelman syndrome presents early in life with profound and severe delays in motor development, language, and cognitive functioning, seizures, and ataxia. And it is a neurodevelopmental disorder that usually does not improve after the onset of symptoms in early childhood, resulting in total dependency on a caregiver. Currently, there are no disease-modifying treatments for Angelman syndrome.
Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and exemptions from certain administrative fees for clinical trials, and seven years of market exclusivity after drug approval. And the FDA defines a rare pediatric disease as a serious or life-threatening disease that primarily affects people from birth to 18 years of age. Under the FDA’s Pediatric Rare 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 for priority review of a marketing application. later 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.
“Receiving FDA Orphan Drug Designation for ION582 reflects the important and urgent need to provide effective treatment for patients living with Angelman syndrome. Ionis is committed to working closely with regulators, clinical researchers, patients and caregivers to advance this new treatment and make it available to those who need it.”
— C. Frank Bennett, Ph.D., Executive Vice President, Chief Scientific Officer and Franchise Leader for Neurological Programs at Ionis
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