The FDA approved Brineura (cerliponase alfa) as a treatment for a specific form of Batten disease. Brineura is the first FDA-approved treatment to slow loss of walking ability (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.
CLN2 disease is an ultra-rare, rapidly progressive fatal brain condition, which affects less than one in one million U.S. residents, many of whom are undiagnosed, BioMarin says. Every year approximately 20 children are born in the U.S. with CLN2 disease.
The drug comes with an enormous price tag, however. According to a Bloomberg article, Brineura will cost $27,000 per biweekly infusion, or about $702,000 a year, and will be one of the most expensive drugs in the world. BioMarin said that most patients who have the disorder are on federal assistance programs like Medicaid, and that the price after mandatory government discounts will be $486,000.
The FDA will require the Brineura manufacturer to further evaluate the safety of Brineura in CLN2 patients below the age of 2, including device related adverse events and complications with routine use. In addition, a long-term safety study will assess Brineura treated CLN2 patients for a minimum of 10 years.
The FDA granted this application Priority Review and Breakthrough Therapy designations. Brineura also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The sponsor is also receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.