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.

What is CLN2 Batten disease?

CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), which may also be collectively referred to as Batten disease. All these disorders affect the nervous system and typically cause worsening problems with vision, movement, and thinking ability.

Who makes Brineura?

San Rafael, Calif. -based BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) approved Brineura (cerliponase alfa) for rare disease late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), which is also called tripeptidyl peptidase 1 (TPP1) deficiency.

How much does Brineura cost?

The cost for Brineura injectable kit (150 mg/5 mL) is around $29,335 for a supply of 1 kits, depending on the pharmacy you visit….Injectable Kit.

Is there a cure for Batten disease?

Eventually, children with Batten disease become blind, wheelchair bound, bedridden, unable to communicate, and lose all cognitive functions. 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).

What is neuronal ceroid lipofuscinosis?

Neuronal ceroid lipofuscinosis (NCL) refers to a group of conditions that affect the nervous system. Signs and symptoms vary widely between the forms but generally include a combination of dementia , vision loss, and epilepsy .

Is Batten disease always fatal?

All types of Batten disease are fatal except adult Batten disease. People who develop symptoms of Batten disease as adults have a normal life expectancy. The name for each type of Batten disease starts with “CLN.” This stands for ceroid lipofuscinosis, neuronal — the name of the affected gene.

Does Batten disease run in families?

Genetics of Batten disease A genetic defect, typically inherited from both parents, causes Batten disease.

What is Batten’s disease life expectancy?

The life expectancy is between ages eight to 10. Juvenile Batten disease occurs in children between ages five and 10. These patients usually live until their late teens or early 20s.

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….

Is neuronal ceroid Lipofuscinoses a rare disease?

Neuronal ceroid lipofuscinosis 3 (CLN3-NCL) is a rare condition that affects the nervous system. Signs and symptoms generally develop between age 4 and 8 years, although later onset cases have been reported.

How long can you live with Batten’s disease?

How is cerliponase alfa used in clinical trials?

In 2012, BioMarin began the first clinical trial on affected patients using their recombinant DNA technology cerliponase alfa which is synthesized using Chinese hamster ovarian (CHO) cell lines. Structure of tripeptidyl peptidase 1 (TPP1) enzyme for which cerliponase alfa is a replacement for.

How many amino acids does cerliponase alfa code for?

Cerliponase alfa is an approximately 59 kDa molecule that codes for 544 amino acids in its proenzyme form while the activated mature enzyme only codes for 368 amino acids. Five amino acid residues have N-linked glycosylation sites.

How does cerliponase alfa bind mannose 6 phosphate receptors?

When the Cerliponase alfa proenzyme reaches target neurons during administration, it binds mannose-6-phosphate receptors on the cell surface to trigger vesicle formation around the receptor-proenzyme complex. The more neutral pH of the cytosol promotes binding of the proenzyme’s M6P targeting sequences to their receptors.