Our Treatments - BioMarin

More than 20 years developing therapies for rare genetic disorders.

Each therapy we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it is designed to address. The result: six first or best-in-class treatments with another four in development.

Aldurazyme^®
(laronidase)

MPS I
Approved
Please see full prescribing information including boxed warning for risk of anaphylaxis.
Naglazyme^®
(galsulfase)

Maroteaux-Lamy
MPS VI
Approved
Please see full prescribing information.
Kuvan^®
(sapropterin dihydrochloride)

PKU
Approved
Please see full prescribing information.
Vimizim^®
(elosulfase alfa)

Morquio A Syndrome
MPS IVA
Approved
Please see full prescribing information including boxed warning for risk of anaphylaxis.
Brineura^®
(cerliponase alfa)

CLN2 Disease
Approved
Please see full prescribing information.
PALYNZIQ^®
(pegvaliase-pqpz) Injection

PKU
Approved
Please see full prescribing information including boxed warning for risk of anaphylaxis.
Vosoritide (BMN 111) Analog of CNP

Achondroplasia
NDA/MAA
⌃
Target Indication

Vosoritide is an investigational analog of C-type Natriuretic Peptide under investigation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.
Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII

Severe Hemophilia A
MAA
⌃
Target Indication

Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.
Learn More about Valoctocogene Roxaparvovec ⌃
BMN 307 AAV
Gene Therapy

PKU
Phase 1
⌃
Target Indication

BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy under investigation to determine if it can normalize blood phenylalanine concentration levels in patients with PKU.
Learn More ⌃
BMN 331 AAV
Gene Therapy

Hereditary Angiodema
(HAE)
Preclinical
⌃
Target Indication

BMN 331 is a gene therapy candidate in development for Hereditary Angioedema.
Learn More about BMN 331 AAV ⌃
BMN 255

Chronic Renal Disease
Pre-clinical
⌃
Target Indication

BMN 255 is an investigational drug candidate in development for chronic renal disease.

Our Products

We find tomorrows.

Each of our approved therapies were driven by the goal of resetting the standard of care for the conditions they treat.

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Our Pipeline

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.

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Compassionate Use Policy

We are driven by patient need.

We are committed to developing safe and effective therapies and providing those therapies to the patients as quickly as possible.

Compassionate Use Policy ⌃