(Bloomberg) -- BioMarin Pharmaceutical Inc. rose in late trading after the company said it sees a path to profitability for its gene therapy for an inherited bleed disorder — in part by reducing investment in the drug.
The company said it will narrow its commercial focus for Roctavian, which treats hemophilia A, to three countries — the U.S., Germany and Italy — where it is approved and reimbursed. BioMarin will cut additional investments in development and manufacturing, according to a statement.
BioMarin said it was “encouraged” by recent progress in helping patients get access to Roctavian at treatment centers and seeks to make the therapy profitable by the end of 2025.
Investors have debated whether BioMarin, under new Chief Executive Officer Alexander Hardy, would continue to support its gene therapy program, given its weak performance so far. Bloomberg Intelligence analysts said Roctavian’s slow launch “represents a significant overhang for BioMarin.” During the quarter, BioMarin said it treated three patients in the U.S. and two in Italy with Roctavian, generating $7 million in revenue.
The biotech firm is relying on the growth of Voxzogo, a drug that treats a type of dwarfism known as achondroplasia, amid disappointing sales of Roctavian. After getting US approval in June 2023, Roctavian, which costs $2.9 million, has reached only a handful of patients.
BioMarin shares rose 3.2% at 4:33 p.m. in extended New York trading. The stock has declined 17% this year through Monday’s close, compared with a 3.1% advance of the S&P 400 Midcap Index.
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