The U.S. Food & Drug Administration (FDA) has approved a drug to fight a form of Batten disease, an extremely rare, progressive and fatal neurological condition that typically leaves children unable to walk or talk by age 6.
The drug is called Brineura, and it’s produced by BioMarin Pharmaceutical, headquartered in San Rafael, Calif.
In a BioMarin news announcement, Brineura was said to specifically slow the “loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.”
About 20 children in the United States are born with this form of Batten disease each year, BioMarin said.
According to the Batten Disease Support & Research Association, Batten disease is actually a group of dozens of lysosomal storage disorders that impact the ability of cells to dispose of waste. Proteins and lipids build up in the cells, which causes a number of neurological symptoms, including loss of motor skills, loss of ambulation, loss of communicative skills, seizures, vision loss and dementia.
Newly approved Brineura is an enzyme replacement therapy and is administered directly to the brain, into the patient’s cerebrospinal fluid.
Along with its approval of Brineura, the FDA gave BioMarin a Rare Pediatric Disease Priority Review Voucher that the company can use to request priority review of a subsequent drug therapy.
Batten disease is genetically based and in some forms, can take many years to present. Batten disease patients are frequently misdiagnosed with seizure disorders, epilepsy or autism.
BioMarin expects to make Brineura available in the United States in June.
