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Seeking to make a difference in rare disease

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.

About BioMarin Australia

Our therapy areas

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Phenylketonuria (PKU)

Phenylketonuria (PKU) is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH).

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Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease

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.

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Mucopolysaccharidosis VI (MPS VI)

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).

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Mucopolysaccharidosis type IVA (MPS IVA)

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).

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Achondroplasia

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.

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Scientific discovery driven by genetic diversity

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.

Read more about our science on BioMarin.com

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Products available in Australia

Please select a product to access further information including the Consumer Medicine Information and Product Information.

Brineura® (cerliponase alfa)

Brineura® is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

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KUVAN® (sapropterin dihydrochloride)

KUVAN® is indicated for the treatment of hyperphenylalaninemia (HPA) by reducing blood phenylalanine levels in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.

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NAGLAZYME® (galsulfase (rch))

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.

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PALYNZIQ® (pegvaliase)

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.

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VIMIZIM® (elosulfase alfa (rch))

VIMIZIM® is indicated for the treatment of MPS IVA  (Mucopolysaccharidosis Type IVA, Morquio A Syndrome).

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VOXZOGO® (vosoritide)

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.

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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.

Treatment is only the beginning

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Patient organisations

For each disease state we seek to treat, there are several organisations and resources that provide great insight and support services.

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Grants and donations

We provide a variety of opportunities to further support patient communities.

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Independent research (For healthcare professionals only)

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.

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Data request (For healthcare professionals only)

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.

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Independent medical education (For healthcare professionals only)

We offer funding to support accredited or non-accredited medical education activities for HCPs regarding BioMarin-controlled or -influenced content.

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There’s something special about BioMarin

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.

View career information at BioMarin.com

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Keep track of our latest investor and media information

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.

Find out more at BioMarin.com

Read our latest press releases

BioMarin contact details

Registered Office

BioMarin Pharmaceutical Australia Pty Ltd
119 Willoughby Rd, Crows Nest, NSW 2065, Australia

Tel: 02 9967 8662
Email: anz@bmrn.com

Place an Order

Registered pharmacies and hospitals only:
Patients and consumers should speak to their community pharmacy or prescriber for more information.

Australia
DHL Direct to Market
Toll Free: 1800 077 421 (Option 1)
Email: d2mcs@dhl.com

New Zealand
Healthcare Logistics
Ph: 09 969 0734
E-mail: orders@healthcarelogistics.co.nz

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Medical Information

Hours: 24 hrs/day, 7 days/week
Email: medinfoasia@bmrn.com

Australia
Toll Free: 1800 387 876
Local: 02 8520 3255

New Zealand
Toll Free: 0800 882 012
Local: 09 801 1051

Adverse Events

Hours: 24 hrs/day, 7 days/week
Email: medinfoasia@bmrn.com
Submit an Adverse Event ›

Australia
Toll Free: 1800 387 876
Local: 02 8520 3255

New Zealand
Toll free: 0800 882 012
Local: 09 801 1051

Report a Product Complaint/Enquiry

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

Clinical Trial Information

For healthcare professionals only:

Email: medinfoasia@bmrn.com

Media Relations

Email: anz@bmrn.com