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Three years of treatment with Brineura (cerliponase alfa) has been shown to effectively and sustainably delay the progression of late childhood Batten disease, compared to its natural course, in an ongoing and long-term extension study, BioMarin announcement.

These new results – focused on motor and language skills demonstrate that Brineura, the first one approved treatment by the United States Food and Drug Administration (FDA) and the European Commission for any form of Batten disease, ccontinues to be a safe and effective therapy for children with its late infantile stage – or CLN2 – disease subtype.

“Every day, week, month and year of maintaining clinical function, including language and mobility, is essential for children with CLN2 disease and their families,” Angela Schulz, MD, principal investigator of the study at the children’s hospital, University Medical Center Hamburg-Eppendorf, Germany, said in a Press release.

“Following these children has allowed us to better understand the effect of treatment over time, which contributes to advancing the quality of care,” she added.

Brineura is an enzyme replacement therapy developed by BioMarin which helps slow the gradual loss of the ability to walk in patients with a type of Batten disease known as CLN2 disease. It is an artificial version of the human enzyme TPP1, which is present at very low levels in this patient population.

To reach cells of the brain and central nervous system, Brineura is given directly into the cerebrospinal fluid that surrounds the brain and spinal cord, using a catheter surgically implanted in the head.

A phase 1/2 open-label trial (NCT01907087), conducted at a site in Ohio and others in Europe, evaluated the efficacy and safety of Brineura 300 mg given every two weeks in children aged 3 to 16 years with CLN2 disease. The 23 patients who completed this 48-week study chose to continue treatment in this 240-week extension study (NCT02485899). Measures of treatment safety and changes in motor and language skills during her 4.8 years are her main goals.

The first results showed that, compared to the 42 patients included in the DEM-CHILD NCL Patient Database – a database used to characterize the natural course of disease severity and progression – treatment with Brineura could prevent the decline of motor and language functions in CLN2 children.

These new data showed the sustainability of the improvements in these skills, with motor and language benefits maintained for more than three years in 19 (83%) of the 23 patients.

After two years of treatment with Brineura, patients’ motor language scores were on average 3.3 points better than those reported for matched children in the Natural History Database. After three years, the scores were on average 3.8 points higher.

In contrast, natural history patients were on average 12 times more likely to experience and maintain a two-point drop in motor language scores after three years compared to patients treated with Brineura..

“CLN2 disease is a destructive brain disease that progresses rapidly in children and data has consistently shown over time that cerliponase alfa can make a big difference in slowing the progression of the disease,” said Hank Fuchs, MD, president of research and development in the world. at BioMarin.

“It is important for us to contribute to the body of medical knowledge on CLN2 disease with data useful to the medical community treating these children,” he added.

The long-term extension study, which ends in July 2020, continues to treat 21 children with 300 mg of Brineura given every two weeks. The two children who left the trial did not do so due to “adverse events,” the statement said.

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