BioMarin Shares Slump as Analysts Cut Targets on Strategic Pivot
Company to divest Roctavian gene therapy after weak sales, raising concerns over its long-term growth strategy and competition for its key drug, Voxzogo.
BioMarin Pharmaceutical (BMRN) shares faced intense pressure this week, trading near 52-week lows as a string of analyst downgrades followed the company’s decision to divest its pioneering gene therapy, Roctavian, amid disappointing sales and concerns over future growth.
The biotechnology firm, a specialist in rare genetic diseases, saw its stock hover around $53.67 in Tuesday trading. The slide was prompted by at least four analyst firms cutting their price targets after BioMarin’s mixed third-quarter earnings report and a significant strategic overhaul.
RBC Capital trimmed its target to $66 from $70, Wells Fargo made a deeper cut to $70 from $90, and Barclays reduced its forecast to $80 from $86. The adjustments reflect mounting investor uncertainty after the company announced it would seek a buyer for Roctavian, a one-time treatment for severe hemophilia A that was once hailed as a potential blockbuster.
The decision to sell Roctavian follows a stark commercial reality. The therapy generated just $3 million in revenue during the third quarter, a fraction of initial expectations. High costs and limited market adoption forced the company to pivot, a move that surprised investors and clouded its long-term outlook.
“The commercial uptake of Roctavian has been slower than we anticipated,” BioMarin CEO Alexander Hardy stated in the company’s recent earnings call, confirming the plan to divest the asset and refocus capital on more promising areas of its pipeline.
While the company posted a 4% year-over-year revenue increase to $776 million for the quarter, it also reported a GAAP net loss of $31 million. More concerning for analysts was the company’s revised future guidance. BioMarin withdrew its specific 2027 revenue target, citing increasing competitive threats to its primary growth driver, Voxzogo, a treatment for achondroplasia, the most common form of dwarfism.
Analysts at RBC Capital cited “increased competition and uncertainty” as the core reasons for their price target reduction. The strategic shift away from gene therapy marks a significant retreat for BioMarin, which invested heavily in Roctavian’s development and regulatory approval. The company will now limit sales to the U.S., Germany, and Italy as it searches for a buyer, according to a report from BioPharma Dive.
The market’s bearish reaction underscores a broader challenge for BioMarin: convincing investors it can generate sustainable growth without Roctavian and with new rivals emerging for Voxzogo. The company is now emphasizing its core enzyme replacement therapies and other pipeline candidates in skeletal conditions to drive future performance.
Despite the recent downgrades and near-term headwinds, a majority of analysts still maintain a “Buy” or “Overweight” rating on BioMarin, with an average price target of around $94, suggesting some on Wall Street believe the sell-off is overdone. They point to the company’s established portfolio and the potential for a more focused, and financially disciplined, strategy following the divestment of the costly gene therapy unit.
Investors will now be closely watching for updates on the Roctavian sale and any further developments on the competitive landscape for Voxzogo, which remains the company’s most critical commercial asset.