FDA Approves First Treatment for a Form of Batten Disease

The US Food and Drug Administration (FDA) on Thursday approved the first-ever treatment for a form of the rare pediatric Batten disease, bringing Biomarin its second lucrative priority review voucher (PRV).

FDA approved Brineura (cerliponase alfa) to slow the loss of walking ability in symptomatic pediatric patients three years old and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

“Brineura is an enzyme replacement therapy. Its active ingredient (cerliponase alfa) is a recombinant form of human TPP1, the enzyme deficient in patients with CLN2 disease. Brineura is administered into the cerebrospinal fluid (CSF) by infusion via a specific surgically implanted reservoir and catheter in the head (intraventricular access device),” FDA explained.

Priority review and breakthrough therapy designations were granted to speed FDA’s review of the treatment, which once approved also came with a PRV as part of the rare pediatric disease PRV program.

This is the second PRV won by Biomarin, which previously won the first-ever PRV and later sold it in 2014 to Regeneron and Sanofi for $67.5 million.

Nine of the last 10 PRVs awarded by FDA have been for rare pediatric disease treatments, while only one has been for a rare tropical disease treatment (PaxVax’s cholera vaccine).

FDA offered another first on Thursday too, with the expansion of the approved use of Bayer’s Stivarga (regorafenib) to include treatment of patients with hepatocellular carcinoma (also known as liver cancer) who have been previously treated with the drug sorafenib. This is the first FDA-approved treatment for a liver cancer in almost a decade, the agency said.