Latebreakers: First drug approved for treatment of phenylketonuria
Sapropterin approved for phenylketonuria treatment
The FDA and Biomarin Pharmaceutical Inc. have announced the approval of sapropterin (Kuvan), the first new molecular entity given a regulatory nod for the treatment of phenylketonuria (PKU), a condition that affects about 50,000 patients worldwide. Sapropterin is the synthetic form of 6R-BH4 (tetrahydrobiopterin), an enzyme cofactor that works with phenylalanine hydroxylase to metabolize phenylalanine. Prolonged exposure to high levels of phenylalanine can result in severe neurological damage. The drug has received seven years of market exclusivity as an orphan drug from the FDA. According to the manufacturer, not all patients with PKU will respond to sapropterin, but a therapeutic trial with the drug can determine who will benefit. Sapropterin should be used in conjunction with a phenylalanine-restricted diet.
Psychiatric Pharmacist Working to Optimize Treatment, Improve Patient Safety
December 13th 2024A conversation with Nina Vadiei, PharmD, BCPP, clinical associate professor in the Division of Pharmacotherapy at University of Texas at Austin College of Pharmacy and a clinical pharmacy specialist in psychiatry at the San Antonio State Hospital.
