Sentynl Therapeutics, Inc.

NULIBRY Shows Significant Survival Benefits in Ultra-Rare MoCD Type A: Landmark Clinical Evidence Published

2 months ago

• Sentynl Therapeutics has published the first comprehensive clinical evidence for NULIBRY (fosdenopterin), showing statistically significant survival benefits in patients with Molybdenum Cofactor Deficiency Type A compared to untreated patients. • The data published in the Journal of Inherited Metabolic Disease revealed untreated patients had 5.1 times higher risk of death, with treated patients also showing significant improvements in cognitive and motor functioning. • MoCD Type A is an ultra-rare genetic disorder affecting approximately 1 in 342,000-411,000 live births, causing toxic sulfite buildup in the brain that leads to irreversible neurological damage if left untreated.

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.