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BioMarin's decision to withdraw Roctavian from the market resulted in charges of about $240 million over the fourth quarter of 2025, including a $119 million inventory write-off. Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, the company is walking away altogether by pulling the treatment from the market. The move follows a “comprehensive effort” to identify a potential buyer for the therapy, BioMarin explained Monday in its fourth-quarter earnings press release. Back in October, the company had decided to divest Roctavian, with CEO Alexander Hardy citing the approach as “consistent with BioMarin's portfolio strategy” and offering “the most promising opportunity for ensuring continued patient access to Roctavian,” he said at the time.  The writing had long been on the wall for BioMarin to part with Roctavian, which was first approved in 2023 as the first one-time gene therapy for hemophilia A. Since mid-2024, the company has left its gene therapy manufacturing plant in an “idle state” as it dwindled its Roctavian operations to solely the U.S., Germany and Italy, three markets where the drug has secured reimbursement policies. At the time, the goal was to cut Roctavian’s direct annual expenses to $60 million starting in 2025 and make the product profitable by the end of 2026.Now, after BioMarin was unsuccessful in finding a “qualified buyer” to take on the product, the company will officially withdraw the med from the market, it said. This decision resulted in charges of about $240 million over the fourth quarter of 2025, including a $119 million inventory write-off and $118 million in asset value impairments.Roctavian sales came out to $36 million in 2025, up from 2024’s $26 million. The drug was still seeing some commercial use in its end of days, with a man in Michigan becoming the state’s first patient to use the therapy outside of clinical trials upon being infused in January, the Detroit Free Press reported earlier this month.Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and Novo Nordisk’s Alhemo. Common gene therapy hurdles, such as eligibility limitations and reimbursement struggles, also pulled Roctavian back. Upon taking the helm in 2023, Hardy had outlined three potential paths for the drug. While the focus at the time was on “establishing the opportunity” for Roctavian, a divestment was listed as Hardy’s third option. BioMarin's post-Roctavian future As BioMarin leaves Roctavian behind, the company is looking to a new chapter after inking its largest-ever transaction late last year with a $4.8 billion acquisition of Amicus Therapeutics. The deal gives BioMarin two potential blockbusters in Fabry disease drug Galafold and Pompe disease combination treatment Pombiliti-Opfolda. The addition of Amicus’ portfolio would “accelerate BioMarin’s revenue growth immediately upon close,” Hardy said at the time. The deal is expected to wrap up in the second quarter of this year. Any contribution from the Amicus products is excluded from BioMarin’s 2026 guidance, which forecasts between $3.3 billion to $3.4 billion in full-year sales. In 2025, the company collected revenue of $3.2 billion, up 13% from 2024. BioMarin attributed much of its yearly growth to market gains for its collection of enzyme therapies, as well as achondroplasia treatment Voxzogo. With $927 million in 2025 sales, Voxzogo is the company’s biggest revenue driver. But William Blair analysts argued in a Tuesday note that BioMarin will ultimately require “additional business development” to fully offset the anticipated Voxzogo erosion to come from new market entrants over the next two years. That competition could come from BridgeBio and its oral candidate infigratinib, which recently showed a statistically significant improvement in annualized height velocity from baseline in a phase 3 trial of children with achondroplasia. Mizuho analysts predicted that BridgeBio’s option could “very well end up being the agent of choice for this indication,” pointing to the wide age group trialed and the convenience of an oral offering. Voxzogo, meanwhile, is administered as a daily subcutaneous injection. However, BioMarin has another achondroplasia option up its sleeve in its early-stage BMN 333, which could be a “potentially superior version of Voxzogo,” an Evercore ISI analyst pointed out after the company revealed positive phase 1 results in August.

BioMarin’s previous CEO wanted to offer money-back guarantees to insurers for covering its hemophilia gene therapy Roctavian. Investors in the company may wish that such a promise on performance could have also included them. For all its blockbuster hopes and high-profile status in the gene therapy field, Roctavian’s commercial saga has ended. After a few months of seeking to divest Roctavian, BioMarin said Monday it has failed to find “a qualified buyer” for the gene therapy and will voluntarily withdraw it from the market. The commercial failure is a reminder of the gap between promising science and a viable drug. Roctavian was never able to find a foothold in the market for severe hemophilia A, a genetic blood-clotting disorder. The drug brought in $36 million in 2025 revenue, up from $26 million the year before — but far less than the company and Wall Street initially hoped . The $12.4 billion biopharma reported a $240 million impairment charge for the fourth quarter related to Roctavian, split roughly evenly between writing off inventory and impairing long-lived assets. BioMarin’s stock $BMRN has dropped about 9.5% over the past year, and is off by more than 45% since early 2023. Shares were down less than 1% at midmorning on Tuesday. Priced at $2.9 million for the single infusion, Roctavian competed with highly effective and established prophylactic treatments like Roche’s Hemlibra and Sanofi’s Altuviiio. While there were initial hopes that the gene therapy may prove to be closer to a cure for hemophilia, the long-term clinical data fell short of that high bar. Treated patients showed gradual declines in the level of factor VIII, a protein that helps clot blood. In BioMarin’s clinical study, one in five Roctavian-treated patients returned to prophylactic treatments after five years. BioMarin’s broader business mirrors the industry’s waning gene therapy enthusiasm over the past decade. A few years ago, the Bay Area company was pushing multiple gene therapy programs ahead against genetic disorders like hereditary angioedema and PKU. Those have since been discontinued, and BioMarin’s earnings call on Monday included zero mentions of gene therapy. Since investing giant Elliott Management took an activist stake in the company in late 2023, BioMarin has narrowed its focus on rare disease treatments. The company brought in new leaders, including former Genentech head Alexander Hardy as its CEO and Roche veteran James Sabry as chief business officer. In December, BioMarin said it would buy Amicus Therapeutics for $4.8 billion, building on top of its existing enzyme therapies business. BioMarin forecast 2026 revenue to fall between $3.33 billion and $3.43 billion, a slight increase from 2025’s total of $3.22 billion. That doesn’t factor in the Amicus deal, which the company expects to close in the second quarter.