BioMarin is providing innovative therapeutics to patients with serious unmet medical needs. All of their commercialized products are for rare genetic disorders.
Ikris Pharma Network is authorized by BioMarin to provide access to their medicines to Indian patients through Named-Patient Import.
BioMarin Products
Vimizim® (elosulfase alfa)
Vimizim® (elosulfase alfa) is the only enzyme replacement therapy (ERT) to address the cause of Morquio A (MPS IVA). People born with Morquio A have a gene that does not work correctly. This gene results in missing or low levels of the N-acetylgalactosamine-6 sulfatase (GALNS) enzyme that breaks down certain complex carbohydrates known as glycosaminoglycans (GAGs). Without sufficient quantities of GALNS, the normal breakdown of GAG is incomplete or blocked and it accumulates in the lysosomes of the cell.
Vimizim at-a-Glance
- Approved in the United States, European Union, Canada, Japan, Australia, Mexico, and Brazil
- Manufactured and commercialized by BioMarin
- Designated as an orphan drug in the United States and the European Union
For more details:
https://www.vimizim.com/v22018/wp-content/uploads/2018/02/Prescribing-Information.pdf
Naglazyme® (galsulfase)
Naglazyme® (galsulfase) is an enzyme replacement therapy for the treatment of Mucopolysaccharidosis VI (MPS VI), an inherited life-threatening lysosomal storage disorder caused by a deficiency of the lysosomal enzyme N-acetylgalactosamine 4-sulfatase, an enzyme normally required for the breakdown of certain complex carbohydrates known as as glycosaminoglycans (GAGs).
Naglazyme at-a-Glance
- First specific approved therapy for the treatment of MPS VI
- Approved in multiple countries
- Designated an orphan drug in the United States and internationally
- Manufactured and commercialized by BioMarin
For full Prescribing Information visit:
Kuvan® (sapropterin dihydrochloride)
Kuvan is the first and only FDA-approved medication for PKU to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU. Kuvan is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. Kuvan is to be used in conjunction with a Phe-restricted diet.
Kuvan at a Glance
- Kuvan Tablets approved in the United States, European Union, Canada, and Japan
- Kuvan Powder approved in the United States
- Designated as an orphan drug in the United States and European Union
- Manufactured and commercialized worldwide with the exception of Kuvan in Japan
For full Prescribing Information click following link
http://kuvan.com/wp-content/file/KUVAN_Prescribing_Information1.pdf.
PALYNZIQ® (pegvaliase-pqpz)
PALYNZIQ® (pegvaliase-pqpz) Injection is the first FDA-approved enzyme substitution therapy designed to address the underlying cause of phenylketonuria (PKU), a rare and genetic brain-threatening condition marked by an inability to break down phenylalanine (Phe). PALYNZIQ is a once-daily self-administered therapy, clinically proven to reduce blood Phe levels in adults with PKU who have uncontrolled blood Phe levels on existing management.
PALYNZIQ At-a-Glance
- PALYNZIQ is the first enzyme therapy approved for adults living with PKU.
- Potential to alter the course of PKU management by limiting the need for a Phe-restricted diet
- Manufactured and commercialized by BioMarin
For Additional Information:
Brineura®(cerliponase alfa)
Brineura® (cerliponase alfa) is the first approved treatment for any form of Batten disease, and is indicated to slow the loss of 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.i CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an approximately estimated 20 children born in the United States each year– less than one in a million Americans
Brineura At-a-Glance
- First approved therapy for any form of Batten disease
- Designated as an orphan drug in the United States and the European Union
- Manufactured and commercialized by BioMarin
For additional information, please click on link or visit the following websites
Voxzogo® (Vosoritide)
Vosoritide (Vosoritide) is a human C type natriuretic peptide (CNP) analog. Vosoritide is a 39 amino acid peptide. Its amino acid sequence includes the 37 C terminal amino acids of the human CNP53 sequence plus Pro Gly on the N terminus to convey resistance to neutral endopeptidase (NEP) degradation. Voxzogo increases linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.
Voxzogo, at a glance:
- Manufactured by BioMarin Pharmaceutical Inc.
- Voxzogo is approved in the United States and European Union.
- Approved to increase linear growth in pediatric patients with achondroplasia.
Please click on the link for additional information or visit the following websites
https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf
Aldurazyme® (Laronidase)
Aldurazyme (Laronidase) is a polymorphic variant of the human enzyme α-L-iduronidase produced by recombinant DNA technology in a Chinese hamster ovary cell line. α -L-iduronidase is a lysosomal hydrolase that catalyzes the hydrolysis of terminal a-L-iduronic acid residues of dermatan sulfate and heparan sulfate. Aldurazyme is used in treating patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms, this medicine has been shown to improve pulmonary function and walking capacity.
Aldurazyme at a glance:
An FDA approved medicine in the United States, European Union.
Manufactured and commercialized by BioMarin.
Approved for treating Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms and this medicine has been shown to improve pulmonary function and walking capacity.
Please click on the link for additional information or visit the following websites
https://www.accessdata.fda.gov/drugsatfda_docs/label/2003/larobio043003LB.pdf
