FDA Expands Indication of BioMarin Pharmaceutical’s Brineura to Include Children with Neuronal Ceroid Lipofuscinosis Type 2 Disease

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The FDA has approved BioMarin Pharmaceutical Inc's Brineura (cerliponase alfa) to treat children under the age of three years with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. According to the company, the expanded indication for Brineura now enables treatment regardless of whether the children are symptomatic or presymptomatic. Previously, the drug was only indicated for symptomatic children over the age of three years with CLN2 disease. The approval was based on positive results from the Phase II 190-203 study.¹

"Today's approval represents a significant step forward in enabling children to be treated with Brineura as early as possible, when we can have the greatest impact in altering the natural course of disease," said Hank Fuchs, MD, president of Worldwide Research and Development at BioMarin. "We know that every day counts for families affected by serious genetic conditions such as CLN2 disease, which is characterized by a rapid onset of neurodegenerative symptoms. We have been working diligently since Brineura’s initial approval to support this expanded use in children of all ages, even before they begin to show symptoms."

The 190-203 trial assessed children for decline in the motor domain of the CLN2 Clinical Rating Scale. The study, which included children between the ages of one and six years, showed a reduced decline in motor function and delayed disease onset in patients administered Brineura. Additionally, results found that children under three years of age maintained normal motor function scores when treated with Brineura. In the eight children who were treated, seven were matched to 18 untreated children from a natural history cohort. In the matched natural history comparators, 11 children (61%) demonstrated an unreversed two-point decline or score of zero by the final assessment.

BioMarin warns against the use of Brineura if a patient experiences active intraventricular access device-related complications, any sign or symptom of acute or unresolved localized infection around the device insertion site, suspected or confirmed central nervous system infection, or shunts used to drain extra fluid around the brain. Further, severe or life-threatening allergic reactions such as anaphylaxis can occur during infusions and up to 24 hours after infusion in patients who have previously received Brineura and those who are taking it for the first time. Other potential adverse events (AEs) include meningitis, device-related complications, cardiovascular AEs, and infusion-associated reactions.

Common AEs include fever, issues with the electrical activity of the heart, decreased or increased protein in the fluid of the brain, vomiting, seizures, device-related complications, hypersensitivity, hematoma, headache, irritability, increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure.¹

"Receiving a CLN2 diagnosis is devastating for families as the disease is life-limiting and can severely impact a child's daily functioning and quality of life from a very young age, with symptoms including seizures, speech and language deficits, impaired movement and vision loss," said Ineka Whiteman, PhD, head, research and medical affairs, Batten Disease Support, Research, & Advocacy Foundation, in the press release. "The opportunity to start Brineura treatment earlier, even before the onset of symptoms, provides newfound hope for the families impacted by this rapidly progressive disease. Importantly, this expanded indication provides further impetus for early diagnosis of CLN2 disease, as we continue advocating for inclusion of CLN2 disease on the (Recommended Uniform Screening Panel) for newborn screening."

Reference

1. U.S. Food and Drug Administration Approves BioMarin's BRINEURA® (cerliponase alfa) for Children Under 3 Years with CLN2 Disease. BioMarin. July 24, 2024. Accessed July 25, 2024. https://investors.biomarin.com/news/news-details/2024/U.S.-Food-and-Drug-Administration-Approves-BioMarins-BRINEURA-cerliponase-alfa-for-Children-Under-3-Years-with-CLN2-Disease/default.aspx