BioMarin Announces Plans to Divest AAV Gene Therapy

SAN RAFAEL, CA — October 28, 2025 — BioMarin Pharmaceutical Inc. announced it is seeking to divest its struggling hemophilia A gene therapy, Roctavian (valoctocogene roxaparvovec). The decision follows persistent commercial challenges and disappointing sales for the one-time treatment. The move marks a pivot back to divestiture, one of the three strategic options laid out by CEO Alexander Hardy after he took the helm.

Low Sales Trigger Portfolio Exit

Roctavian, which carries a list price of $2.9 million, generated only $3 million in third-quarter sales. Total sales for the year stand at $23 million. The therapy faces intense competition in the hemophilia A market from blockbuster treatments like Roche’s Hemlibra and Sanofi’s Altuviiio, alongside eligibility and reimbursement hurdles common to gene therapies.

“We continue to believe Roctavian has an important role to play in the treatment of hemophilia A and are therefore evaluating out-licensing options for this innovative gene therapy,” CEO Alexander Hardy stated in an earnings press release. “This decision is consistent with BioMarin’s portfolio strategy and offers the most promising opportunity for ensuring continued patient access to Roctavian.”

Reversal of Strategy

The decision to divest reverses a strategy implemented last year, when BioMarin opted to downsize the drug’s commercial footprint. Since August 2024, the company had focused Roctavian efforts solely on its three secured reimbursement markets: the U.S., Germany, and Italy. This earlier restructuring also involved ceasing further life-cycle development and placing the gene therapy manufacturing plant in an “idle state.”

At the time, BioMarin hoped to cut the drug’s annual expenses to $60 million and achieve profitability by the end of 2026.

While the company explores out-licensing opportunities, Roctavian will remain available in the U.S., Germany, and Italy, and BioMarin will continue to provide support and monitoring for already-treated patients.

Analysts were largely unfazed by the announcement, with Evercore ISI calling the move “a bit anti-climactic at this point.” BioMarin now shifts its commercial focus to its achondroplasia treatment, Voxzogo, which is expected to contribute between $900 million and $935 million toward the company’s projected full-year revenues of $3.15 billion to $3.2 billion.