Shortly after taking the reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling hemophilia A gene therapy, Roctavian. A year after opting to shrink the drug’s commercial reach, BioMarin has now flipped back to option C: a divestiture.
After booking $3 million in third-quarter sales for the $2.9 million drug, the company has decided to pursue “options to divest Roctavian and remove it from our portfolio,” Hardy said in an Oct. 27 earnings press release.
“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,” Hardy explained. “This decision is consistent with BioMarin's portfolio strategy and offers the most promising opportunity for ensuring continued patient access to Roctavian.”
While it explores out-licensing opportunities, the company will still make the drug available in its three markets the—U.S., Germany and Italy—and “continue to provide support and monitoring” for those already treated, it said.
Analysts were largely unfazed by the move, with Evercore ISI calling it “a bit anti-climactic at this point” in a recent note to clients.
Although Hardy last April said the company was focused on “establishing the opportunity” and “not engaging with people around the divestment of Roctavian,” the drugmaker's eventual decision to limit the product's commercial reach may have spelled the beginning of the end.
Since August of last year, BioMarin has focused its Roctavian efforts solely on the U.S., Germany and Italy, three markets where the drug has secured reimbursement policies. The move also meant that no further life-cycle development activities were on the books, and the company’s gene therapy manufacturing plant was put into an “idle state” until additional doses were needed.
At the time, BioMarin hoped to cut Roctavian’s direct annual expenses to $60 million starting in 2025 and make the product profitable by the end of 2026. In 2024, BioMarin logged $26 million in total Roctavian sales.
Consistent sales struggles
The gene therapy competes with a variety of treatments for hemophilia A, including Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and Novo Nordisk’s Alhemo, which was recently approved as a once-a-day treatment for hemophilia A or B. The range of other treatment options, along with eligibility limitations and reimbursement hurdles for gene therapies, made broad uptake an uphill battle.
So far this year, the therapy has drummed up total sales of $23 million, according to BioMarin’s third-quarter earnings report.
As it looks to rid itself of the hemophilia A med, BioMarin remains focused on achondroplasia treatment Voxzogo and its collection of enzyme therapies. The company expects full-year revenues to total between $3.15 billion and $3.2 billion, with Voxzogo contributing between $900 million and $935 million.
The third quarter saw $776 million in product revenue for BioMarin, a 4% increase from the same quarter last year.