Who makes Brineura?
Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2020. 5.
Is Brineura a biologic?
Brineura is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 disease. It is an enzyme replacement therapy designed to restore TPP1 enzyme activity and break down the storage materials that cause CLN2 disease….
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What is Brineura used for?
Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (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.
Is Brineura an orphan drug?
Because the number of patients with CLN2 is low, the disease is considered ‘rare’, and Brineura was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 March 2013.
Where is Brineura approved?
Brineura is the first approved treatment by the U.S. Food and Drug Administration and the European Commission for any form of Batten disease. Brineura is also approved in Scotland, England, and Wales.
How much does Brineura cost?
Brineura will carry a list price of $27,000 per carton, with patients requiring one carton every other week, according to BioMarin execs. That’s a total of $702,000 per year before any discounts.
Is Batten disease treatable?
There is no cure for these disorders but a treatment for one of the forms (CLN2 disease) has been approved by the U.S. Food and Drug Administration (see Treatment section). Children with all forms of Batten disease have a greatly shortened life expectancy.
Is there a cure for Batten disease?
The disorder affects the body’s ability to get rid of cellular waste (lipids and proteins), so they build up in cells all over the body. The buildup causes seizures, vision loss, problems with thinking and movement, and eventually, death. There is no cure for Batten disease.
What is Batten’s Disease?
Batten disease is a group of fatal genetic disorders. There are 13 types. The disorder affects the body’s ability to get rid of cellular waste (lipids and proteins), so they build up in cells all over the body. The buildup causes seizures, vision loss, problems with thinking and movement, and eventually, death.
How long can you live with Batten disease?
Children with Batten disease have a greatly shortened life expectancy. Children with infantile Batten disease often die in early childhood. Children with later onset forms of the disease may live into their teens to thirties, while those who develop the disease in adulthood may have a normal life expectancy.