Posted by Michael Wonder on 27 Apr 2017
FDA approves first treatment for a form of Batten disease
27 April 2017 - The U.S. FDA today 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 paediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.
The FDA will require the Brineura manufacturer to further evaluate the safety of Brineura in CLN2 patients below the age of 2 years, 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 Paediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare paediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the tenth rare paediatric disease priority review voucher issued by the FDA since the program began.
