Aug. 31 at 8:03 PM
$FBIO “Its CUTX-101 (copper histidinate) treatment is on track for FDA approval by its September 30, 2025, PDUFA date as the first therapy for Menkes disease, a rare disorder where untreated patients typically die by age 3 due to severe copper deficiency. Clinical trials show early treatment reduces mortality risk by nearly 80%, extending median survival to 177.1 months versus 16.1 months for untreated controls (p<0.0001), with most untreated patients succumbing before age 3. 2624 The drug also improves neurodevelopmental outcomes and is well-tolerated, with no treatment-related serious adverse events in 66 patients. 27 FDA’s Breakthrough Therapy, Fast Track, and Orphan Drug Designations, plus priority review, underscore strong regulatory support for addressing this unmet need. With compelling data and no reported issues, CUTX-101 is highly likely to gain approval, offering hope to prevent early death in Menkes patients.”
