BioMarin receives notice of allowance for Kuvan patent

BioMarin Pharmaceutical Inc. has announced that it has received a notice from the United States Patent Office reporting allowance of claims covering once daily dosing methods for Kuvan (sapropterin dihydrochloride) in the treatment of phenylketonuria (PKU).

The company expects that the patent will be officially issued later this year, and if issued, the patent's initial 20-year term would expire in 2024. The company has a number of other pending patent applications covering various aspects of Kuvan compositions and dosing.

"We believe the issuance of this patent will be significant in strengthening our proprietary position on Kuvan," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "This patent would prevent potential competitors from using or copying Kuvan's approved dosing regimen. Based on the claims allowed by the USPTO, we believe that the once daily dosing regimen will prevent therapeutically equivalent generic competition to Kuvan while the patent is in force."

Kuvan (sapropterin dihydrochloride) Tablets are indicated in the United States 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.

The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe.

Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan has received seven years of orphan exclusivity in the United States and ten years of market exclusivity in the E.U.

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase. PAH is required for the metabolism of phenylalanine, 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 mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth.