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.
Pharmacists Play Unique Role in Advancing Health Equity for Patients With Chronic Disease
December 7th 2023A new study, outlined in a poster at ASHP Midyear 2023, identified 3 key themes associated with the ways in which pharmacists are positioned to advance health equity for patients with chronic diseases.
Assessing Financial Impacts of Pharmacist-Led Interventions in Pediatric Ambulatory Care
December 5th 2023According to a poster abstract presented at the ASHP Midyear Clinical Meeting & Exhibition, specialty pharmacy services were used for a variety of conditions that have been reported in adult populations.
