FDA approves first treatment for a form of Batten disease

The US Food and Drug Administration today approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease.

Rare disease drug developer BioMarin Pharmaceutical’s (Nasdaq: BMRN) Brineura is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients three years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. Every year around 20 children are born in the USA with CLN2 disease. These affected children completely lose the ability to walk and talk around six years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult with death often occurring between eight and 12 years of age.

“The FDA is committed to approving new and innovative therapies for patients with rare diseases, particularly where there are no approved treatment options,” said Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, adding: “Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition.”