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FDA Extends Market Exclusivity of KUVAN Powder for Oral Solution and Tablets

Mon, 04/14/2014 - 8:22am

BioMarin Pharmaceutical today announced that the 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.

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