BioMarin Announces Approval of Brineura
June 12, 2017
BioMarin is incredibly proud to announce the approval and launch of Brineura™, an enzyme-replacement therapy for an ultra-rare pediatric brain disorder called neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura™ was approved in the US on April 27, 2017, with European approval following on June 1.
CLN2, a form of Batten Disease, is an ultra-rare, rapidly progressive fatal brain condition which affects less than one in one million US residents, many of whom are undiagnosed. Every year approximately 20 children are born in the US with CLN2 disease. These affected children completely lose the ability to walk and talk around 6 years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult, with death typically occurring between 8 and 12 years of age. Brineura™ It is the first-ever enzyme replacement therapy to be directly administered into the brain, treating the underlying cause of the condition by replacing the deficient enzyme in patients.
BioMarin is investing in tools and resources to educate physicians on CLN2 in order to help them diagnose patients earlier, preventing misdiagnosis during critical years when therapy could help slow the loss of brain function and ambulation. To provide additional support to families of children with neurologic disorders, BioMarin is also offering a no-cost genetic testing program called “Behind the Seizure.” This program will support early testing for children who experience seizures, one of the first manifestations of CLN2.
The full US press release is available here.
CLN2, a form of Batten Disease, is an ultra-rare, rapidly progressive fatal brain condition which affects less than one in one million US residents, many of whom are undiagnosed. Every year approximately 20 children are born in the US with CLN2 disease. These affected children completely lose the ability to walk and talk around 6 years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult, with death typically occurring between 8 and 12 years of age. Brineura™ It is the first-ever enzyme replacement therapy to be directly administered into the brain, treating the underlying cause of the condition by replacing the deficient enzyme in patients.
BioMarin is investing in tools and resources to educate physicians on CLN2 in order to help them diagnose patients earlier, preventing misdiagnosis during critical years when therapy could help slow the loss of brain function and ambulation. To provide additional support to families of children with neurologic disorders, BioMarin is also offering a no-cost genetic testing program called “Behind the Seizure.” This program will support early testing for children who experience seizures, one of the first manifestations of CLN2.
The full US press release is available here.
