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.