There's a New Treatment for Batten Disease

The US Food and Drug Administration (FDA) has approved the first treatment for a specific form of Batten disease.

This newly approved medication is called Brineura (cerliponase alfa). The FDA approved it to help slow the loss of walking ability in patients who have tripeptidyl peptidase-1 (TPP1) deficiency, also known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). It was approved for use in patients who are 3 years old and older.

CLN2 disease is one of a group of conditions referred to as Batten disease. It's a rare, inherited disease that affects the nervous system. Children with this disease may have language delays and seizures at the start of the disease. Later, they may experience muscle twitches and vision loss. CLN2 disease makes it difficult to sit and walk.

Brineura is available as an injection. A health care professional administers it once every other week into the cerebrospinal fluid (CSF). CSF is the fluid that surrounds the brain and spinal cord.

Commonly reported side effects include fever, vomiting, seizures, headache and electrocardiogram abnormalities like a slow heart rate.

BioMarin Pharmaceutical manufactures Brineura.