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In April 2017, the drug was approved to treat the slow loss of the ability to walk for symptomatic patients aged 3 and older who had Batten disease.
Updated at 5:00 pm on July 24, 2024
The FDA has approved a supplemental biologics license application for cerliponase alfa (Brineura; BioMarin Pharmaceuticals) indicated to slow the loss of ambulation for children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also referred to as tripeptidyl peptidase 1 deficiency. In April 2017, the drug was approved to treat the slow loss of the ability to walk for symptomatic patients aged 3 and older who had Batten disease, according to an article published on Pharmacy Times.1,2
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"Today's approval represents a significant step forward in enabling children to be treated with [cerliponase alfa] 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, in a news release. "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 [cerliponase alfa’s] initial approval to support this expanded use in children of all ages, even before they begin to show symptoms.”1
The sBLA was supported by data from Study 190-203, a phase 2 open-label, multicenter trial that evaluated the drug over approximately 3 years for children aged 1 through 6 years at baseline, including 8 children younger than 3 years. Previously, in 2019, the drug demonstrated a reduction in decline for patients with CLN2 disease compared to the untreated group for 3 years. Response to treatment was approximately 83%, with patients treated with the drug having an average 3.8 better motor-language score compared to those who were untreated. Decline was defined as having a sustained 2-point loss in motor score by week 169.1,3
In the current analysis, among the 8 children below age 3, 0 had a 2-point decline or score of 0 in motor score by week 169. Further, among the matched individuals in the control group, 61% experienced an unreversed 2-point decline or score of 0 at the final assessment. The data were presented at the 20th Annual We’re Organizing Research on Lysosomal Disease meeting in February.1
Trial Name: A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
ClinicalTrials.gov ID: NCT02678689
Sponsor: BioMarin Pharmaceutical
Completion Date: April 2022
"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," Ineka Whiteman, PhD, head of research and medical affairs at the Batten Disease Support, Research, & Advocacy (BDSRA) Foundation, said in the news release. "The opportunity to start [cerliponase alfa] 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."1
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