Investor Elliott reportedly takes $1bn stake in BioMarin

Armed with a recent FDA approved single-dose hemophilia A gene therapy, BioMarin Pharmaceutical has reportedly received interest from activist investor Elliott Management.  

According to Reuters, two people “familiar” with the situation have claimed investment management firm Elliott has built a stake in BioMarin following months of discussions about the firm’s future.

The unnamed sources said the hedge fund has around $60 billion in assets and has invested more than $1 billion on the rare genetic disease focused firm, BioMarin. However, they could not outline any demands the hedge fund may have put forward.

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BioMarin has had various wins over the last year, including approval from the US Food and Drug Administration (FDA) for its single-dose hemophilia A gene therapy, Roctavian (valoctocogene roxaparvovec) in July. Last month, the agency also expanded Voxzogo’s (vosoritide) label to include children younger than five with Achondroplasia. Voxzogo is a C-type natriuretic peptide (CNP), which is administered once a day via injection.

Additionally, the firm’s Q3 earnings call reported a total revenue of $581 million, which represents 15% growth year-on-year. However, despite an increase in revenue and FDA approvals, BioMarin discussed the challenges associated with launching Roctavia.

“It has taken several months in the US following approval to align stakeholders and to facilitate first patients treated,” Jeffrey Ajer, chief commercial officer at BioMarin told shareholders during the call.

“This is a result of that unique profile of one-time reimbursement and treatment with Roctavian. Navigating hospital administration and finance adds a level of complexitiy for hemophilia treatment centers or HTCs […] while time consuming our one-tome events are essential to facilitate patient access going forward. In short, the groundwork completed in 2023 is expected to enable simplified access to Roctavian [in] 2024.”

Elliott swooped in to support contract development manufacturing organization (CDMO) Catalent in August. As part of the deal, the investor group is responsible for establishing a “Strategic and Operational Review Committee to conduct a review of the Company’s business, strategy and operations, as well as the Company’s capital-allocation priorities, in order to maximize the long-term value of the Company.”