Cyprium Therapeutics, Inc.

FDA Grants Priority Review to CUTX-101 for Menkes Disease, Offering Hope for Rare Pediatric Condition

5 months ago

• The FDA has accepted Sentynl Therapeutics' NDA for CUTX-101, granting priority review for the treatment of Menkes disease, a rare genetic disorder. • Clinical trials of CUTX-101 demonstrated an almost 80% reduction in mortality risk compared to untreated patients, significantly improving overall survival. • CUTX-101 has been granted multiple designations, including Breakthrough Therapy and Orphan Drug, highlighting its potential to address a critical unmet need. • Cyprium Therapeutics is eligible to receive up to $129 million in milestone payments and royalties, retaining ownership of a potential Priority Review Voucher.

FDA Grants Priority Review for CUTX-101 in Treatment of Menkes Disease

a year ago

The U.S. FDA has accepted for review and granted Priority Review to the New Drug Application for CUTX-101, a potential treatment for Menkes disease, with a target action date set for June 30, 2025. CUTX-101 has shown promising clinical efficacy, significantly improving overall survival in patients with this rare pediatric disease.