News|Videos|July 28, 2025

WATCH: Experts react to the FDA approval of sepiapterin to treat PKU

Name: WATCH: Experts react to the FDA approval of sepiapterin to treat PKU
Uploaded: 2025-07-29T01:15:01.556Z
Duration: 6 min 28 s
Description: Suzanne Hollander, MS, RD, LDN and Timothy Tramontana, MD offer thoughts on the newly FDA-approved sepiapterin for PKU.

Author(s)Suzanne Hollander, MS, RD, LDN, Timothy Tramontana, MD

Fact checked by: Joshua Fitch, Senior Editor

Suzanne Hollander, MS, RD, LDN; and Timothy Tramontana, MD, offer thoughts on newly FDA-approved sepiapterin for PKU.

On July 28, 2025, the FDA approved PTC Therapeutics' sepiapterin (Sephience) to treat adult and pediatric patients with phenylketonuria (PKU). Sepiapterin is now the third FDA-approved medication for lowering blood phenylalanine (Phe) levels and can be used alongside a Phe-restricted diet to expand protein tolerance in individuals with sepiapterin-responsive PKU.