Study: Drug Therapy Reduced Rate of Decline in Children with CLN2
The study was aimed at the efficacy and safety of cerliponase alfa (Brineura, BioMarin) for patients with CLN2.
An ongoing open-label extension study found that cerliponase alfa (Brineura, BioMarin) reduced the rate of decline in patients with neuronal ceroid lipofuscinosis type 2 (CLN2) compared to an untreated group for 3 years, as measured by the CLN2 Clinical Rating Scale.
The study, held in 5 centers across Europe, was aimed at assessing the efficacy and safety of intraventicular cerliponase alfa in children with CLN2, also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease. Eligible patients were CLN2 patients, aged 3 years to 16 years, with a combined score of 3-6 for the motor language dimensions on the CLN2 scale.1
A response to treatment was seen in approximately 83% of treated patients after 3 years. This was determined by whether or not there was an unreversed 2-point decline in the motor-language (ML) scale or a score of 0, since such outcomes represent clear and substantial motor and language decline.
At 3 years, untreated patients were found to be 12 times more likely than treated patients to have a 2-point decline in the ML score. After 3 years with cerliponase alfa therapy, treated patients' ML scores were on average 3.8 points better than untreated, whereas after 2 years of treatment these patients had an average of 3.3 points more than natural history, according to the press release.
"Every day, week, month and year of maintaining clinical function, including language and mobility, is critical to children with CLN2 disease and their families," said Angela Schulz, MD, lead study investigator, in a prepared statement. "Following these children has allowed us to better understand the effect of the treatment over time, which contributes to the advancement in the standard of care."
According to Genetics Home Reference, CLN2 disease is an inherited disorder that affects the nervous system beginning around ages 2 years and 4 years. CLN2 initially features symptoms such as recurrent seizures and difficulty coordinating movements, and affects motor and language development, such as sitting, walking and speech.2
Cerliponase alfa was approved in April 2017 by the FDA to slow the loss of ambulation in symptomatic pediatric patients aged 3 years and older with late infantile CLN2, and is the first therapy approved to treat children with CLN2 disease. The drug also was approved in June 2017 by the European Commission.
- BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years. https://www.prnewswire.com/news-releases/biomarin-announces-ongoing-study-demonstrates-durable-treatment-benefit-from-brineura-cerliponase-alfa-for-3-years-300792075.html. Published Feb. 07, 2019. Accessed Feb. 14, 2019.
- CLN2 disease. Genetics Home Reference. https://ghr.nlm.nih.gov/condition/cln2-disease. Accessed Feb. 14, 2019.