The FDA has granted accelerated approval to Avlayah (tividenofusp alfa-eknm) for the treatment of neurologic manifestations of Hunter syndrome in pediatric patients, marking the first new therapy for the rare genetic disorder in nearly 20 years.
Avlayah is indicated for children weighing at least 5 kilograms with either presymptomatic or symptomatic disease prior to advanced neurologic impairment, according to a March 25 news release from Denali Therapeutics. It is the first FDA-approved enzyme replacement therapy designed to cross the blood-brain barrier and treat neurologic symptoms of mucopolysaccharidosis Type 2.
In a clinical trial of 47 pediatric patients, the therapy lowered a key disease marker in spinal fluid by 91% at 24 weeks. At that point, most children had levels similar to those seen in individuals without the disease. The most common side effects included infusion-related reactions, infections, fever, anemia and rash.
Avlayah is administered once weekly for pediatric patients weighing at least 5 kilograms prior to advanced neurologic impairment.
Denali was awarded a Rare Pediatric Disease Priority Review Voucher alongside the approval, according to a March 26 FDA news release. A confirmatory phase 2/3 trial, Compass, is underway and includes young adult participants.
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