BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in $600 million annually.
BioMarin Pharmaceutical is purchasing Amicus Therapeutics in an all-cash deal worth $4.8 billion, in a bid to pump up its rare disease portfolio after a series of cuts.
The deal prices shares of the New Jersey–based Amicus at $14.50, a 33% premium on the company’s last closing value, according to BioMarin’s announcement Friday morning. Shares of BioMarin are up 4.6% to $54.34 in pre-market trading Friday.
The acquisition brings two FDA-approved drugs to BioMarin’s rare disease offerings: Galafold, an oral treatment for Fabry disease, and Pombiliti/Opfolda, a two-part therapy for Pompe disease. Amicus also brings the U.S. rights to DMX-200, a Phase III small molecule treatment for focal segmental glomerulosclerosis, a rare and fatal kidney disease.
BioMarin hopes the Amicus buy will “accelerate revenue growth” for the company. Over the past year, Galafold and Pombiliti/Opfolda together generated $599 million in total sales for Amicus, according to the deal announcement.
The transaction will be a shot in the arm for BioMarin if those sales figure hold. In fall 2024, BioMarin promised investors $4 billion in annual revenue by 2027, without accounting for potential mergers and acquisitions.
By October of this year, the company had abandoned that hope, cutting revenue guidance for its achondroplasia therapy Voxzogo. That treatment was one of the company’s top sellers in 2024, with $735.1 million in revenue. It fell short only to Vimizim, BioMarin’s mucopolysaccharidosis treatment, which brought in $739.8 million for the year, according to the company’s annual report.
When announcing the $4 billion goal miss, BioMarin revealed the divestiture of hemophilia A treatment Roctavian, citing stagnant sales. Roctavian was supposed to be a major leg of the company’s revenue target. After a restructuring effort announced in late 2024, BioMarin reorganized itself into three units, one of which focused solely on Roctavian.
Cutting Roctavian was the second portfolio prune for BioMarin in recent months. In August, the company cut a preclinical phenylketonuria asset.
Amicus is the second major acquisition for BioMarin this year, after the purchase of enzyme replacement company Inozyme for $270 million in May. The revenue prospects from that purchase are a bit further out. Inozyme’s lead molecule, INZ-701 for treating the rare, progressive disease ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency, just wrapped a Phase I/II trial late last year and is headed into late-stage development.
