Tuesday, April 7, 2026

BioMarin’s Presence in Uncommon Enzyme Issues Grows With $4.8B Amicus Therapeutics Acquisition

Uncommon illness drug developer BioMarin Pharmaceutical is shopping for its solution to income development with a $4.8 billion deal for Amicus Therapeuticsan organization whose two commercialized merchandise every supply blockbuster potential and in addition complement their acquirer’s lengthy historical past in uncommon enzyme deficiency therapies.

The deal introduced Friday is the second acquisition for BioMarin below 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 alternative 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 therapy for achondroplasia, the most typical type of dwarfism. Permitted 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 improvement), as a result of now we have a compelling set of capabilities and now we 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 capability to compete with Voxzogo in achondroplasia. However we will do many issues on the similar time and we will create extraordinary worth, we consider, for all of our stakeholders by doing offers akin to 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 might pursue choices to divest the gene remedy. The Amicus acquisition continues BioMarin’s technique of embracing its strengths in uncommon enzyme deficiencies. In Could, it agreed to pay $270 million to accumulate Inozyme Pharmawhose lead program is in late-stage improvement 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 in regards to the Amicus merchandise.

Amicus was based in 2005 by John Crowley, an entrepreneur who was pursuing new remedies for 2 of his youngsters 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% enhance 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 convey a base of income that’s rising at a quicker fee than BioMarin’s portfolio. The corporate tasks every Amicus product will change into a blockbuster vendor. Hardy added that the money move from the enterprise mixture will allow BioMarin to construct up its monetary reserves inside 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 word 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 almost $2 billion by 2035. Schwartz added that whereas there are few particulars on synergies and value financial savings, generally the enterprise mixture “seems very logical primarily based on the strategic and monetary overlap.”

“Now we have lengthy heard from the corporate relating to their sturdy and rising enzyme remedy enterprise, so the acquisition of Amicus makes good 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 firms 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.

Photograph illustration: Piotr Swat/SOPA Photographs/LightRocket, through Getty Photographs

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