Fortress Biotech (NASDAQ:FBIO) and its majority-owned subsidiary, Cyprium Therapeutics, secured a historic regulatory victory Tuesday as the U.S. Food and Drug Administration approved ZYCUBO® (copper histidinate) as the first-ever treatment for Menkes disease.
The approval of ZYCUBO marks a paradigm shift for the treatment of Menkes disease, a rare and typically fatal genetic disorder that severely impairs copper absorption, leading to progressive neurodegeneration.
Clinical data underpinning the approval showed that early intervention with the subcutaneous injection resulted in a staggering 80% reduction in the risk of death.
Patients treated early achieved a median overall survival of 177.1 months (approximately 14.7 years), compared to just 17.6 months for untreated historical controls.
Under the terms of a strategic asset purchase agreement, Sentynl Therapeutics—a subsidiary of Zydus Lifesciences—will take over the commercialization of ZYCUBO.
The milestone triggers a series of lucrative financial vents for Cyprium, including eligibility for up to $129 million in development and sales milestones, alongside tiered royalties.
Most notably, a Rare Pediatric Disease Priority Review Voucher (PRV) will be issued to Cyprium upon the approval; such vouchers have recently commanded market prices exceeding $100 million in secondary sales.
Menkes disease affects approximately 1 in 100,000 live births, primarily in males.
Without treatment, children rarely survive past the age of three.
With ZYCUBO’s approval, the companies plan to immediately pivot toward global access initiatives and newborn screening programs to ensure the earliest possible diagnosis and treatment.