A new drug from Ionis Pharmaceuticals has become the first to be approved by the FDA for treatment of a rare brain disorder, the drugmakers shared on Thursday.
The injectable treatment zilganersen, branded as Zanvastro, can now be used in adults and children with Alexander disease, a rare, progressive genetic disorder of the central nervous system that damages brain cells and impacts movement, speech and swallowing.
There is no cure for the condition, which usually begins in early childhood and affects fewer than 1,000 people in the US according to the National Institute of Health, and around 1 in 3 million people worldwide.
In an early to late-stage study, the drug was seen to significantly improve the walking ability of patients with Alexander disease.
In the 54-patient trial, those that received a 50mg dose of zilganersen showed a statistically significant increase in gait speed as assessed by a 10-meter walk test at 61 weeks.
The treatment stems the effects of the disease by blocking the production harmful brain protein, GFAP, which builds up abnormally in patients with the disease due to a genetic mutation, contributing to a decline in white matter of the brain and the general nervous system.
It is administered to patients through the spinal canal by a healthcare professional once every three months and will now be available to patients across America, with Ionis partnering with Italian drugs company Recordati in the hope to make it available across Europe in the future.
Analysts predict that the drug could generate peak annual sales of $295m.
Brett P. Monia, Chief Executive Officer, Ionis, said: “Today’s approval of Zanvastro (zilganersen) begins a new chapter for people living with Alexander disease and their families, who have long faced this relentlessly progressive and often fatal disease with no treatment options.”
“This transformative approval also marks our first independent launch from our industry-leading neurology pipeline and underscores the power of our RNA-targeted technology to address serious neurological diseases without adequate treatment options,” he added.
Dr Amy Waldman, paediatric neurologist and lead investigator for the Zanvastro (zilganersen) study, Children’s Hospital of Philadelphia, said: “For decades, care for people living with Alexander disease has focused primarily on managing symptoms, without an option to modify the underlying cause of disease.”
She said that the introduction of the drug signified “a significant advancement in care”, adding: “For the first time, we can move beyond managing individual manifestations of the disease to addressing its underlying biology, with the potential to meaningfully improve outcomes for this community.”
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