Drug offers hope for children with rare brain disease
The Food and Drug Administration has approved a drug that can be used to treat a rare pediatric brain disorder in children known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a BioMarin Pharmaceutical Inc. release said.
The release said Brineura (cerliponase alfa) has been approved to help patients 3 years old and older who have CLN2, a form of Batten disease. It is a fatal brain condition that affects less than 1 in 1 million. However, many who suffer from it are undiagnosed. There are about 20 children born in the U.S. each year that have CLN2.
The motor skills of these children decline so rapidly that by the time they are 6 years old they can no longer walk or talk, the release said. As it progresses feeding and other every day needs become difficult. Most patients die between 8 and 12 years of age.
Brineura is the first treatment approved for children with CLN2 disease, the release said.
Brineura works by slowing the loss of ambulation. It is an enzyme replacement therapy that replaces the deficient TPP1 enzyme and is administered directly to the brain.
"The approval of Brineura is an extraordinary medical breakthrough for the CLN2 Batten community who have been waiting for this moment for more than a century when the condition was first described," Margie Frazier, executive director of Batten Disease Support and Research Association, said in the release.
Brineura was approved in under four years from starting the first clinical trial to today, a significant achievement for a condition that progresses so rapidly, BioMarin Chairman and CEO Jean-Jacques Bienaime said in the release.
Organizations in this story
BioMarin Pharmaceuticals 770 Lindaro St San Rafael, CA 94901
U.S. Food and Drug Administration (FDA) 10903 New Hampshire Ave Silver Spring, MD 20903