What is CLN2 disease?
CLN2 disease is an inherited disorder that primarily affects the nervous system. The signs and symptoms of this condition typically begin between ages 2 and 4. The initial features usually include recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia).
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….
| Contacts: | |
|---|---|
| Investors | Media |
| (415) 455-7558 | (415) 455-7451 |
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.
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.
How long can you live with Batten’s disease?
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.
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.
| Quantity | Per unit | Price |
|---|---|---|
| 1 | $29,335.46 | $29,335.46 |
How long do you live with Batten’s Disease?
What is Batten’s disease life expectancy?
What are treatments for Batten Disease?
No specific treatment is known that can reverse the symptoms of any form of Batten disease. In 2017 the Food and Drug Administration approved an enzyme replacement therapy for CLN2 disease (TTP1 deficiency) called cerliponase alfa (Brineura®) that has been shown to slow or halt the progression of symptoms.
What are the signs and symptoms of Batten disease?
The first signs of Batten disease include:
- Vision loss (this symptom does not affect adults with Batten disease).
- Epilepsy (seizures).
- Cognitive problems, trouble learning or difficulty keeping up in school.
- Problems with speaking.
- Clumsiness and issues with coordination, balance and movement.
Is Batten disease neurological disorder?
Batten disease, also known as neuronal ceroid lipofuscinosis, refers to a group of rare inherited neurological conditions that can cause vision loss, progressive motor and cognitive decline, and seizures.
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.
What do you need to know about Cln2 disease?
What is CLN2 disease? CLN2 disease is a rare genetic disorder that affects children. 1,2 Through ongoing research, doctors have made important discoveries that are helping us understand why this disease happens. How can I provide the best care for my child?
How is Brineura used to treat Cln2 disease?
Brineura is the first enzyme replacement therapy to be directly administered into the fluid of the brain, treating the underlying cause of CLN2 disease by helping to replace the deficient TPP1 enzyme missing in affected children.
What makes BioMarin Pharmaceuticals different from other drug companies?
Treatment is only the beginning. We are committed to the communities we treat beyond the therapies we develop. We take on the biggest challenges in rare disease. Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
Why are rare diseases important to BioMarin Pharmaceutical?
Our focus on rare disease gives us the rare opportunity to truly impact lives. We are inspired and driven everyday by stories like Sheri’s. Treatment is only the beginning. We are committed to the communities we treat beyond the therapies we develop.
