FDA Extends Market Exclusivity Six Months for BioMarin's Rare Disease Therapy KUVAN(R) (sapropterin dihydrochloride) Powder for Oral Solution and Tablets

FDA Extends Market Exclusivity Six Months for BioMarin's Rare Disease Therapy KUVAN(R) (sapropterin dihydrochloride) Powder for Oral Solution and Tablets

New Convenient Form of KUVAN Now Available for the Treatment of Phenylketonuria (PKU)

SAN RAFAEL, Calif., April 14, 2014 (GLOBE NEWSWIRE) -- BioMarin
Pharmaceutical Inc. (Nasdaq:BMRN), a global leader in the development
and commercialization of therapies for rare genetic diseases, today
announced that the Food and Drug Administration (FDA) granted KUVAN(R)
(sapropterin dihydrochloride) Powder for Oral Solution and Tablets a
six-month pediatric exclusivity extension. The FDA action extends
KUVAN's market exclusivity to June 2015 and is based on studies
submitted in response to a written request by the FDA to investigate
the use of KUVAN in pediatric patients from birth to age 6. KUVAN
formulated as Tablets and Powder for Oral Solution, is the first and
only FDA-approved medication for PKU to reduce blood phenylalanine
(Phe) levels in patients with hyperphenylalaninemia (HPA) due to
tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). KUVAN is
to be used in conjunction with a Phe-restricted diet.

PKU or PAH Deficiency is a rare genetic condition in which the body
cannot process Phe, an amino acid found in many foods. This can lead to
too much Phe in the blood. Left untreated, high blood Phe levels can
affect the brain, impair thinking, and cause behavioral problems.

The FDA has approved a new and convenient form of KUVAN, a powder for
oral solution packaged in individual packets of 100 mg. KUVAN powder is
to be dissolved in water, apple juice or a small amount of soft foods.
This new formulation is a convenient alternative to tablets and
provides an additional option for infants and small children. KUVAN
powder dissolves rapidly and completely, and offers reduced acidity.

"Seven years after the approval of KUVAN Tablets, BioMarin remains
committed to the PKU or PAH deficient community. The new powder form of
KUVAN provides an additional option for parents with small children
taking KUVAN, or for anyone who has trouble swallowing tablets," said
Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We
continue to invest in the PKU community with the introduction of this
new solution and to advance another experimental PKU treatment with PEG
PAL, a therapy in Phase 3 clinical development."

In January 2014, the American College of Medical Genetics and Genomics
(ACMG) issued new practice guidelines, which support the need for
lifelong management of PHE levels in patients with phenylketonuria or
PKU. The new diagnosis and management guidelines provide the first
update to recommendations for therapy of PKU since the 2001 National
Institutes of Health Consensus statement.1

The new guidelines state that treatment of PKU should be initiated as
early as possible and must be continued throughout adulthood and for
life. Patients treated from the early weeks of life with initial good
metabolic control, but who lose that control in later childhood or
adult life, may experience both reversible and irreversible
neuropsychiatric consequences. The guidelines specifically note that
for appropriate patients use of KUVAN should be considered to help
lower Phe.

About KUVAN(R)

First approved in December 2007, KUVAN is the first and only
prescription medicine of its kind. When used in conjunction with diet,
KUVAN can help lower blood Phe levels more than the use of diet alone.
The newly revised American College of Medical Genetics and Genomics
(ACMG) PAH-deficiency management guidelines (2013) specifically
recommend trial therapy with KUVAN to assess patient responsiveness.

KUVAN (sapropterin dihydrochloride), formulated as Tablets and Powder
for Oral Solution, 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 Powder for Oral Solution is available immediately. For more
information about KUVAN, please visit www.KUVAN.com.

Important Safety Information

KUVAN(R) (sapropterin dihydrochloride) Tablets and KUVAN (sapropterin
dihydrochloride) Powder for Oral Solution are approved to reduce blood
Phe levels in patients with hyperphenylalaninemia (HPA) due to
tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is
to be used with a Phe-restricted diet.

High blood Phe levels are toxic to the brain and can lead to lower
intelligence and decrease in the ability to focus, remember and
organize information. Any change you make to your diet may impact your
blood Phe level. Follow your doctor's instructions carefully. Your
doctor and dietitian will continue to monitor your diet and blood Phe
levels throughout your treatment with KUVAN.

If you have a fever, or if you are sick, your Phe level may go up. Tell
your doctor and dietitian as soon as possible so they can see if they
have to adjust your treatment to help keep your blood Phe levels in the
desired range.

KUVAN is a prescription medicine and should not be taken by people who
are allergic to any of its ingredients. Tell your doctor if you have
ever had liver or kidney problems, are nursing or pregnant or may
become pregnant, have poor nutrition or are anorexic. Your doctor will
decide if KUVAN is right for you. Tell your doctor about all the
medicines you take.

The most common side effects reported when using KUVAN are headache,
diarrhea, abdominal pain, upper respiratory tract infection (like a
cold), throat pain, vomiting, and nausea.

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical
Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.

KUVAN is available by prescription only. To learn more, please visit
www.kuvan.com for full prescribing information. If you have any
questions about this information, please talk with your doctor.

About PKU or PAH Deficiency

Phenylketonuria (PKU) or phenylalanine hydroxylase (PAH) deficiency is
a genetic disorder affecting approximately 50,000 diagnosed patients in
the developed world and is caused by a deficiency of the enzyme
phenylalanine hydroxylase (PAH), this enzyme is required for the
metabolism of phenylalanine (PHE), an essential amino acid found in
most protein-containing foods. If the active enzyme is not present in
sufficient quantities, PHE accumulates to abnormally high levels in the
blood and becomes toxic to the brain, resulting in a variety of
complications including severe intellectual disability, seizures,
tremors, behavioral problems and psychiatric symptoms. As a result of
newborn screening efforts implemented in the 1960s and early 1970s,
virtually all individuals with PKU or PAH deficiency under the age of
40 in developed countries are diagnosed at birth and treatment is
implemented soon after. PAH deficiency can be managed with a
PHE-restricted diet, which is supplemented by low-protein modified
foods and PHE-free medical foods; however, the strict diet is difficult
for most patients to adhere to the extent needed for achieving adequate
control of blood PHE levels. KUVAN, the first and only prescription
medicine of its kind, may help individuals with PAH deficiency lower
blood PHE levels when used in conjunction with a PHE-restricted diet,
more than the use of diet alone. To learn more about PAH deficiency,
please visit www.PKU.com. Information on this website is not
incorporated by reference into this press release. Some of the signs
and symptoms of high blood PHE include:


--  For infants and children: severe intellectual disability and
     developmental delay, skin rash (eczema), light-colored skin, eyes and
     hair (hypopigmentation)
--  For teens and adults: lower intelligence, psychological and psychiatric
     symptoms like anxiety, depression and phobias, problems with memory and
     performing tasks (executive function), poor concentration and irritable
     mood among other things.
--  For pregnant women: increased risk for the baby's growing brain,
     including risk of intellectual disability, increased risk for a small
     head (microcephaly) and other problems such as a heart malformation
     (congenital heart defect) and poor overall growth (intrauterine growth
     retardation). This teratogenic effect of PHE on the developing fetus is
     called Maternal PKU syndrome.


About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product
portfolio comprises five approved products and multiple clinical and
pre-clinical product candidates. Approved products include VIMIZIM(TM)
(elosulfase alfa) for MPS IVA; Naglazyme(R) (galsulfase) for MPS VI;
Aldurazyme(R) (laronidase) for MPS I, a product which BioMarin
developed through a 50/50 joint venture with Genzyme, a Sanofi Company;
KUVAN(R) (sapropterin dihydrochloride) Powder for Oral Solution and
Tablets, for phenylketonuria (PKU), developed in partnership with Merck
Serono, a division of Merck KGaA of Darmstadt, Germany and Firdapse(R)
(amifampridine), which has been approved by the European Commission for
the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product
candidates include PEG PAL (PEGylated recombinant phenylalanine ammonia
lyase), which is currently in Phase 3 clinical development for the
treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP)
inhibitor, which is currently in Phase 3 clinical development for the
treatment of germline BRCA breast cancer, BMN 701, a novel fusion
protein of insulin-like growth factor 2 and acid alpha glucosidase
(IGF2-GAA), which is currently in Phase 1/2 clinical development for
the treatment of Pompe disease, BMN 111, a modified C-natriuretic
peptide, which is currently in Phase 1 clinical development for the
treatment of achondroplasia, BMN 190, a recombinant human tripeptidyl
peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal
ceroid lipofuscinosis (CLN2), a form of Batten Disease, which is
currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the
treatment of hemophilia A and BMN 250, a novel fusion of
alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from
insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB.

For additional information, please visit www.BMRN.com.

Vimizim(TM) is our trademark, and BioMarin(R), Naglazyme(R), KUVAN(R),
Firdapse(R) are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.

1https://www.acmg.net/docs/Phenylalanine_Hydrosylase_Deficiency_Practic
e_Guideline_AOP_Jan_2013.pdf


CONTACT: Investors
        Traci McCarty
        BioMarin Pharmaceutical Inc.
        (415) 455-7558

        Media
        Debra Charlesworth
        BioMarin Pharmaceutical Inc.
        (415) 455-7451

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