BioMarin Undergoes Strategic Restructuring Amid Slow Roctavian Sales and Staff Layoffs

Restructuring Efforts:
BioMarin has initiated a strategic restructuring, including laying off 225 employees, which represents 7% of its global workforce, to address slow sales of its hemophilia gene therapy, Roctavian.

Roctavian Sales:
Despite being the first marketed gene therapy for the most common form of hemophilia, Roctavian has seen disappointing sales, with net revenue reaching only $800,000 in the first quarter of 2024 and $3.5 million for all of 2023, significantly below initial estimates.

Pipeline Review: BioMarin has discontinued four experimental therapies that did not meet its thresholds for patient impact and commercial opportunity, focusing instead on accelerating three other programs:
BMN 333 for multiple growth disorders, BMN 349 for AATD-associated liver disease, and BMN 351 for Duchenne muscular dystrophy.

Cost Savings:
The company expects to save $50 million to $60 million this year by discontinuing the four pipeline programs, though some of these savings will be reinvested in the accelerated programs.

Future Plans:
BioMarin aims to complete its transformation by the end of the year and has proposed reducing investments in Roctavian development and manufacturing, which is expected to lower annual direct costs to around $60 million starting next year.