BioMarin Pharmaceutical has announced the withdrawal of Roctavian, the first one-time gene therapy for hemophilia A, after failing to secure a qualified buyer. This strategic exit resulted in approximately $240 million in charges during the fourth quarter of 2025, covering inventory write-offs and asset impairments.
Despite its landmark approval in 2023, Roctavian struggled with commercial uptake due to high costs, complex reimbursement hurdles, and stiff competition from established treatments like Hemlibra. The therapy generated only $36 million in revenue throughout 2025, a figure insufficient to sustain its specialized manufacturing operations and direct expenses.
Moving forward, BioMarin is shifting its focus toward a new growth chapter, highlighted by its $4.8 billion acquisition of Amicus Therapeutics. The company aims to accelerate revenue through rare disease treatments for Fabry and Pompe diseases while striving to maintain the market dominance of Voxzogo (for achondroplasia) amid the emergence of new competitors in 2026.