By Sriparna Roy
Sept 17 (Reuters) – The U.S. Food and Drug Administration on Thursday approved Ultragenyx Pharmaceutical’s gene therapy, making it the first approved treatment for a rare and fatal childhood disease.
The therapy, branded as Fayuvi, was approved for pediatric patients with Sanfilippo syndrome Type A, an inherited condition that progressively damages the brain and nervous system.
The disease is caused by a deficiency of an enzyme called sulfamidase, leading to an abnormal accumulation of a complex sugar molecule known as heparan sulfate in the body and brain. This toxic buildup causes children to gradually lose cognitive, speech, and motor skills.
Ultragenyx shares were up over 10% in afternoon trading.
Administered as a single intravenous infusion, the gene therapy delivers a functional copy of the SGSH gene into cells, enabling the body to produce sulfamidase and clear the damaging buildup.
In trials, children treated with the therapy maintained or improved cognitive function compared with untreated patients, according to the FDA.
Glenn O’Neill, president and co-founder of nonprofit Cure Sanfilippo Foundation, whose daughter Eliza was diagnosed in 2013, said the approval means “real hope and a real life-changing chance to have a different outcome than the traditional course of this disease, a better future for their children, a better quality of life.”
Eliza was treated with the therapy in 2016, when she was 6 years old.
“There’s been significant benefits that we’ve seen over the years,” Eliza’s parents told Reuters ahead of the decision.
“She’s never had a seizure, she is not on a feeding tube, she is not in a wheelchair, she goes to school, she swims in the pool … we have seen those kind of differences grow in the abilities and quality of life our daughter has been able to maintain versus what would be expected for this disease.”
Ultragenyx expects Fayuvi to be available at specialized U.S. treatment centers within 30 to 60 days.
(Reporting by Sriparna Roy and Kamal Choudhury in Bengaluru; Editing by Maju Samuel, Shilpi Majumdar and Tasim Zahid)
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