The FDA has expanded the approval of BioMarin’s treatment for a genetic neurological condition that spurs seizures and twitching in young children.
The agency said Brineura can be used in children of all ages, regardless of whether they’ve begun to experience symptoms of the disease, BioMarin announced Wednesday. The treatment was previously approved for kids ages 3 or older who had symptoms of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease.
The broader label was more than six years in the making after the treatment became the first ever to snag FDA approval for the disease in 2017. Signs of the condition often arise in kids ages 2 to 4, and the FDA’s action should meaningfully expand the number of patients who can be treated.
“We have been working diligently since Brineura’s initial approval to support this expanded use,” BioMarin president of global R&D Hank Fuchs said in a statement.
The therapy has not been a core revenue driver for BioMarin, a likely reflection of how rare the condition is. Previous estimates say the disease incidence is less than 0.5 per 100,000 live births.
It brought in $161.9 million in 2023, a 5% increase from 2022, and sales were essentially flat in the first quarter of this year.
But it’s good news for a company that has reshaped itself under new CEO Alexander Hardy. In May, BioMarin announced layoffs of 170 employees, which were slated to be completed by the end of this month. The drug developer has also grappled with sluggish sales of its hemophilia A treatment, which brought in just $3.5 million in 2023 and $800,000 in the first quarter of 2024.