BioMarin Pharma plans to divest struggling gene therapy

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(Reuters) -BioMarin Pharmaceutical said on Monday it plans to divest its gene therapy, a product once expected to be a blockbuster treatment for a type of rare bleeding disorder.

The company said it is exploring out-licensing options for the therapy, Roctavian, and will remove it from its portfolio. BioMarin will continue to make the therapy available in the U.S., Germany and Italy.

Roctavian was approved in the U.S. in 2023 to treat adults with severe hemophilia A, a rare genetic disorder where the blood doesn’t clot properly because the body lacks a protein called factor VIII.

But the launch struggled due to limited patient eligibility, slow reimbursement, and a high price tag of $2.9 million. The therapy generated just $3.5 million in sales in 2023 and about $26 million in 2024.

BioMarin had initially projected Roctavian would bring in $100 million to $200 million in 2023, but later cut that forecast to $50 million to $100 million, and eventually to less than $10 million.

The weak performance drew the attention of activist investor Elliott Investment Management, which took a stake of over $1 billion in the company in November 2023, according to a Reuters report.

Elliott reached a deal with BioMarin to appoint three new independent directors and form a strategic review committee to evaluate the company’s operations.

BioMarin said it will now focus on its more profitable business units, including enzyme therapies and skeletal conditions.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Maju Samuel)