SAN RAFAEL, Calif., Feb. 6, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (BMRN) appear today that The WORLDSymposium 2018 awarded Brineura® (cerliponase alfa) the New Analysis Award, which recognizes important achievements in ameliorative advancements for lysosomal diseases. The accolade was presented on Monday, February 5.
BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)
“We are accustomed that Brineura has been accustomed by the WORLDSymposium as the aboriginal accustomed analysis to amusement a anatomy of Batten disease,” said Hank Fuchs, M.D., President of Accepted Research and Development at BioMarin. “The approval of Brineura was a absolute absorption of an abiding charge and affiliation amid families, advocates and physicians. We allotment this accolade with those communities and attending advanced to continuing to accommodate our abutment to beforehand the affliction of accouchement with CLN2 disease.”
Brineura is the alone analysis accustomed by the U.S. Food and Drug Administration and European Union to amusement accouchement with backward baby neuronal ceroid lipofuscinosis blazon 2 (CLN2), additionally accepted as tripeptidyl peptidase 1 (TPP1) deficiency. In the U.S., Brineura is accustomed to apathetic the accident of airing in accouchement over 3 with backward baby CLN2. CLN2 ache is an ultra-rare, rapidly accelerating baleful academician condition, which affects an estimated 1,200 to 1,600 accouchement worldwide. Afflicted accouchement aboriginal appearance affection about the age of 3 and absolutely lose the adeptness to airing and allocution about 6 years of age. During the afterwards stages of the disease, agriculture and disposed to accustomed needs become actual difficult with afterlife usually occurring amid 8 and 12 years of age.
Brineura was additionally awarded the 2017 Popular Science “Best of What’s New” accolade in the bloom category. Each year, Popular Science reviews bags of new articles and innovations beyond 11 categories for its anniversary “Best of What’s New” issue, selecting those that represent a cogent footfall advanced in their category.
This is additionally the added time BioMarin has accustomed the WORLDSymposium New Analysis Award. BioMarin accustomed the accolade in 2015 for the aboriginal and alone analysis accustomed to amusement mucopolysaccharidosis blazon IVA, additionally accepted as Morquio A syndrome.
About CLN2 Disease
Children with CLN2 ache about activate experiencing seizures amid the ages of 2 and 4 years old, preceded in the majority of cases by accent development delay. The ache progresses rapidly with best afflicted accouchement accident the adeptness to airing and allocution by about 6 years of age. Initial affection are followed by movement disorders, motor deterioration, dementia, blindness, and afterlife usually occurring amid the ages of 8 and 12 years of age. During the afterwards stages of the disease, agriculture and disposed to accustomed needs become actual difficult. BioMarin estimates the accident of CLN2 ache is about one in 200,000 with up to 1,200 to 1,600 accouchement in the regions of the apple area BioMarin operates, abounding of whom are undiagnosed.
The neuronal ceroid lipofuscinoses (NCLs) are a amalgamate accumulation of lysosomal accumulator disorders that includes the autosomal backward neurodegenerative ataxia CLN2 disease. CLN2 ache is acquired by mutations in the TPP1 gene consistent in amiss action of the agitator tripeptidyl peptidase 1 (TPP1). In the absence of TPP1, lysosomal accumulator abstracts commonly metabolized by this agitator accrue in abounding organs, decidedly in the academician and retina. Buildup of these accumulator abstracts in the beef of the afraid arrangement accord to the accelerating and adamant neurodegeneration which manifests as accident of cognitive, motor, and beheld functions.
Brineura is a recombinant anatomy of animal tripeptidyl peptidase 1 (TPP1), the agitator amiss in patients with CLN2 disease. It is an agitator backup analysis advised to restore TPP1 agitator action and breach bottomward the accumulator abstracts that account CLN2 disease. In adjustment to adeptness the beef of the academician and axial afraid system, the analysis is delivered anon into the aqueous surrounding the academician (cerebrospinal fluid) application BioMarin’s patented technology.
For added admonition apropos this product, amuse acquaintance BioMarin Medical Admonition at [email protected]
IndicationBrineura® (cerliponase alfa) is a decree medication acclimated to apathetic accident of adeptness to airing or clamber (ambulation) in adapted pediatric patients 3 years of age and earlier with backward baby neuronal ceroid lipofuscinosis blazon 2 (CLN2), additionally accepted as tripeptidyl peptidase 1 (TPP1) deficiency.
Important Safety InformationBrineura is a decree medicine. Afore analysis with Brineura, it is important to altercate your child’s medical history with their doctor. Tell the doctor if they are ailing or demography any medication and if they are allergic to any medicines. Your child’s doctor will adjudge if Brineura is adapted for them. If you accept questions or would like added admonition about Brineura, acquaintance your child’s doctor.
Brineura is alone accustomed by beverage into the aqueous of the academician (known as an intraventricular injection) and application antiseptic address to abate the accident of infection. An intraventricular admission accessory or anchorage charge be in abode at atomic 5 to 7 canicule above-mentioned to the aboriginal infusion. Intraventricular admission device-related infections were empiric with Brineura treatment. If any signs of infection occur, acquaintance your child’s doctor immediately. Your child’s intraventricular admission accessory may charge to be replaced over time.
Brineura should not be acclimated in patients with alive intraventricular admission device-related complications (e.g., leakage, accessory failure, or device-related infection) and with shunts acclimated to cesspool added aqueous about the brain.
Low claret burden and/or apathetic affection amount may action during and afterward the Brineura infusion. Acquaintance your child’s doctor anon if these reactions occur.
Undesirable or hypersensitivity reactions accompanying to Brineura treatment, including fever, vomiting, and irritability, may action during analysis and as backward as 24 hours afterwards infusion. Your adolescent may accept medication such as antihistamines afore Brineura infusions to abate the accident of reactions. Austere and astringent allergic reactions (anaphylaxis) may occur. If a acknowledgment occurs, the beverage will be chock-full and your adolescent may be accustomed added medication. If a astringent acknowledgment occurs, the beverage will be chock-full and your adolescent will accept adapted medical treatment. If any signs of anaphylaxis occur, anon seek medical care.
Safety and capability in pediatric patients beneath 3 years of age accept not been established.
The best accepted ancillary furnishings appear during Brineura infusions included fever, problems with the electrical action of the heart, decreased or added protein in the aqueous of the brain, vomiting, seizures, hypersensitivity, accumulating of claret alfresco of claret argosy (hematoma), headache, irritability, and added white claret corpuscle calculation in the aqueous of the brain, device-related infection, apathetic affection rate, action jittery, and low claret pressure. Intraventricular device-related ancillary furnishings included infection, commitment system-related complications, and added white claret corpuscle calculation in aqueous of the brain.
These are not all of the accessible ancillary furnishings with Brineura. Allocution to your child’s doctor if they accept any affection that bother them or that do not go away.
Call your doctor for medical admonition about ancillary effects. You may address ancillary furnishings to FDA at 1-800-FDA-1088.
Please see accompanying abounding Prescribing Information, or visit www.Brineura.com.
About BioMarinBioMarin is a all-around biotechnology aggregation that develops and commercializes avant-garde therapies for bodies with austere and life-threatening attenuate disorders. The company’s portfolio consists of six commercialized articles and assorted analytic and pre-clinical artefact candidates.
For added information, amuse visit www.BMRN.com.
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
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