Uncommon illness drug developer BioMarin Pharmaceutical is shopping for its approach to income development with a $4.8 billion deal for Amicus Therapeuticsan organization whose two commercialized merchandise every provide blockbuster potential and likewise complement their acquirer’s lengthy historical past in uncommon enzyme deficiency therapies.
The deal introduced Friday is the second acquisition for BioMarin underneath CEO Alexander Hardy, the Genentech veteran who joined the corporate two years in the past. At the moment, BioMarin envisioned new development would come from the hemophilia A gene remedy, Roctavian. However lackluster gross sales of the product have led the corporate to pivot, turning to M&A to construct out its pipeline and portfolio.
BioMarin’s first commercialized product was an enzyme substitute remedy and the San Rafael, California-based biotech at the moment markets 5 such merchandise. The portfolio is led by a more moderen product, Voxzogo, a remedy for achondroplasia, the most typical type of dwarfism. Authorized in 2021, this peptide drug has change into BioMarin’s prime vendor. However looming competitors on this indication from Ascendis Pharma and BridgeBio Pharma is placing strain on BioMarin to search out different methods to develop income.
“We’re doing this (acquisition), as we stated, with regard to (enterprise growth), as a result of we now have a compelling set of capabilities and we now have the skills to do offers and create worth for our shareholders,” Hardy stated throughout a Friday morning convention name. “On the similar time, we’re very assured of our skill to compete with Voxzogo in achondroplasia. However we are able to do many issues on the similar time and we are able to create extraordinary worth, we consider, for all of our stakeholders by doing offers reminiscent of this one.”
For the 9 months ended Sept. 30, BioMarin reported $2.3 billion in income. Roctavian accounted for simply $23 million of that whole. In October, BioMarin introduced it will pursue choices to divest the gene remedy. The Amicus acquisition continues BioMarin’s technique of embracing its strengths in uncommon enzyme deficiencies. In Might, it agreed to pay $270 million to amass Inozyme Pharmawhose lead program is in late-stage growth for an enzyme deficiency that at the moment has no FDA-approved therapies. When that deal was introduced, Hardy stated the Inozyme remedy will profit from BioMarin’s expertise figuring out eligible sufferers to develop gross sales for its enzyme deficiency therapies. On Friday, he stated the identical factor concerning the Amicus merchandise.
Amicus was based in 2005 by John Crowley, an entrepreneur who was pursuing new remedies for 2 of his kids born with the uncommon enzyme deficiency Pompe illness. The biotech’s first commercialized product was Galafold, a drug permitted in 2018 for a distinct enzyme deficiency named Fabry illness. In 2023, Amicus gained FDA approval for Pombiliti and Opfolda, a two-drug routine for Pompe. Mixed, these Amicus merchandise accounted for $448.9 million in income within the first 9 months of 2025, an 18.5% improve in comparison with the identical interval in 2024.
BioMarin Chief Monetary Officer Brian Mueller stated that regardless of the dimensions of the acquisition, the deal doesn’t include any scientific trial threat and the 2 commercialized Amicus merchandise deliver a base of income that’s rising at a quicker price than BioMarin’s portfolio. The corporate tasks every Amicus product will change into a blockbuster vendor. Hardy added that the money stream from the enterprise mixture will allow BioMarin to construct up its monetary reserves throughout the subsequent 12 to 24 months to deploy towards extra offers. Within the meantime, he stated BioMarin will discover smaller offers to construct out its R&D pipeline.
The Amicus acquisition is predicted to instantly speed up BioMarin’s income development, Leerink Companions analyst Joseph Schwartz stated in a observe despatched to traders. The agency estimates the 2 commercialized Amicus merchandise will tally $630 million in 2025 income, rising to greater than $1 billion by 2027 and practically $2 billion by 2035. Schwartz added that whereas there are few particulars on synergies and price financial savings, generally the enterprise mixture “appears very logical primarily based on the strategic and monetary overlap.”
“We have now lengthy heard from the corporate concerning their sturdy and rising enzyme remedy enterprise, so the acquisition of Amicus makes excellent sense to us,” he stated.
The acquisition phrases introduced Friday name for BioMarin to pay $14.50 in money for every Amicus share, which is a 33% premium to Thursday’s closing value and a 58% premium to the inventory’s common value for the previous 60 days. BioMarin stated it plans to finance the transaction with money readily available and about $3.7 billion in debt financing. The boards of administrators of each corporations have permitted the deal, which nonetheless wants the approval of Amicus shareholders and regulators. The businesses count on to finish the transaction within the second quarter of 2026.
Picture illustration: Piotr Swat/SOPA Pictures/LightRocket, by way of Getty Pictures
