Strategic Shifts for BioMarin’s Gene Therapy Roctavian Amid Market Challenges
May 2, 2024
BioMarin’s gene therapy for hemophilia A, Roctavian, received approval in Europe in 2022 and the U.S. in 2023, but has unfortunately seen limited patient uptake, treating only four individuals to date. The therapy generated modest sales, with the most recent figures showing $800,000 from one patient in Italy with a total of $3.5 million in 2023. The company cites reimbursement and market access issues as major barriers preventing wider adoption. To improve sales from this therapy, BioMarin is concentrating efforts in the U.S., Italy, and Germany, where reimbursement approvals already exist. The company’s new CEO, Alexander Hardy, outlined three potential futures for Roctavian: continuing as is if patient uptake increases significantly, adjusting the level of investment in the therapy, or considering a divestiture if it fails to meet expectations. The decision on Roctavian’s future, including possible divestiture, will be detailed at BioMarin’s investor day in September.
Amidst these market access issues, BioMarin is shifting its focus towards the dwarfism drug Voxzogo, which generated $152 million in the first quarter alone, a 70% increase year-over-year. This strategic pivot highlights the company’s broader approach to prioritize the most productive assets amidst ongoing challenges.
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[Source: FiercePharma, April 30 2024]
