03/26/2026
Big progress in the PKU world 👏
With new treatments like repinatrabit showing promising results, we’re entering a new era—one where more individuals may be able to move toward controlled, flexible protein intake instead of strictly limiting everything.
At PKU Perspectives, we’re getting ready for that future.
Whether someone is:
• Strict low protein
• Transitioning on new medications
• Exploring a 2–5g flexible protein approach
—we are working to support you at every step along the way.
Our goal is to grow with this community and provide real food solutions for every stage of the PKU journey.
💙 Progress in treatment means progress in nutrition—and we’re evolving right alongside you.
Otsuka America Pharmaceutical, Inc. shared positive results from a long-term, open-label extension study of repinatrabit (JNT-517), an investigational drug, in teens with PKU at the American College of Medical Genetics and Genomics (ACMG) meeting.
In this study, teens took a low dose of repinatrabit twice a day. After one month of treatment, their blood phenylalanine (Phe) levels dropped by an average of 67%. All teen participants showed a response to the treatment, including both responders and non-responders to sapropterin (Kuvan®). No serious adverse events were reported, and the safety profile was consistent with what has been seen in adults.
Repinatrabit is continuing to be evaluated in children and is currently being tested in adults with PKU in the Phase 3 PheORD clinical trial in order to achieve approval by the Food and Drug Administration.
Read the full press release: https://tinyurl.com/4cxphz8z
This is an exciting step toward bringing another treatment option to people with PKU!
