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news.The US Food and Drug Administration (FDA) has granted BioMarin Pharmaceutical's Kuvan (sapropterin dihydrochloride) Powder for Oral Solution and Tablets a six-month pediatric exclusivity extension.
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FDA action extends Kuvan’s market exclusivity to June 2015 and is based on trials submitted in response to a written request by the FDA to investigate the use of the drug in pediatric patients from birth to age six.
The drug 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).
The company said that 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.
FDA has approved a new and convenient form of Kuvan, a powder for oral solution packaged in individual packets of 100mg.
The new formulation is a convenient alternative to tablets and provides an additional option for infants and small children.
BioMarin chief executive officer Jean-Jacques Bienaimé said seven years after the approval of Kuvan Tablets, the company 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," Bienaimé said.
"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."