We seek the big effect
Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.
At BioMarin, we view ourselves as a team of pioneers, willing to take risks in the quest to find new solutions to address serious, debilitating challenges in rare diseases, and motivated by the desire to help improve the lives of patients with unmet medical needs.
Achondroplasia is a rare genetic bone growth disorder and the most common form of disproportionate short stature. It is caused by a change in the fibroblast growth factor receptor 3 (FGFR3) gene, which impairs the growth of bone in the cartilage of the growth plate.
Phenylketonuria (PKU) is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH).
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease is an ultra-rare and rapidly progressive paediatric brain disorder and one of the most common forms of neuronal ceroid lipofuscinosis, a group of inherited disorders collectively known as Batten disease.
Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy Syndrome, is an ultra-rare inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B), an enzyme required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs).
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an ultra-rare inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine-6-sulfatase (GALNS), an enzyme required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs).
BioMarin is committed to translating genetic discoveries into transformative medicines. Our approach focuses on diseases with precisely understood mechanisms that are rooted in GENETICS, developing TARGETED therapeutic interventions that address the underlying causes of diseases, utilising the readily ACCESSIBLE endpoints that are proximal to the disease pathway, and enabling TRANSFORMATIONAL changes to the way patients feel, function, and survive.
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BRINEURA® is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
KUVAN® is indicated for the treatment of hyperphenylalaninemia (HPA) by reducing blood phenylalanine levels in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
NAGLAZYME® is indicated for the treatment of Mucopolysaccharidosis VI (MPS VI) storage disorder, a disease in which the enzyme level of Nacetylgalactosamine 4-sulfatase is absent or lower than normal.
PALYNZIQ® is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 and older, whose blood phenylalanine levels cannot be adequately controlled by other means such as by diet.
VIMIZIM® is indicated for the treatment of MPS IVA (Mucopolysaccharidosis Type IVA, Morquio A Syndrome).
VOXZOGO® is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.
For each disease state we seek to treat, there are several organisations and resources that provide great insight and support services.
We provide a variety of opportunities to further support patient communities.
We provide product and funding support for independent research in rare diseases and genetic disorders to unleash scientific innovation and meet the unmet medical needs.
We seek to improve patient care and drive advancement in medical science, through providing qualified scientific researchers access to de-identified patient-level data collected in clinical trials.
We offer funding to support accredited or non-accredited medical education activities for HCPs regarding BioMarin-controlled or -influenced content.
Hope. It’s where BioMarin’s story begins. It’s what we work to deliver every day. As a biopharmaceutical company, our success stems from our unwavering focus on science and the hope our therapies can bring to patients and their families. Explore our story, and let our opportunities speak to you.
Transparency creates trust. As a listed biopharmaceutical company, BioMarin is obligated to enhance transparency of our operation with readily available information. BioMarin enables you to keep abreast of our latest development and events, track the NASDAQ index, and get access to other company-related useful information. You will be impressed by how much we have devoted to achieving corporate responsibility, with the aim of boosting efficiency and reducing the environmental impacts.
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Email: anz@bmrn.com
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Australia
Toll Free: 1800 387 876
Local: 02 8520 3255
New Zealand
Toll free: 0800 882 012
Local: 09 801 1051
Registered pharmacies and hospitals only:
Australia
DHL Direct to Market
Toll Free: 1800 077 421 (Option 3)
Email: d2mcs@dhl.com
New Zealand
Healthcare Logistics
Ph: 09 969 0734
Email: orders@healthcarelogistics.co.nz
Email: anz@bmrn.com
Email: ir@bmrn.com
Tel: +1 (415) 455-7558