Valoctocogene roxaparvovec

BioMarin plans to dish out $4.8 billion to acquire Amicus Therapeutics and its two approved rare disease medicines, the California biotech said Friday. By gaining access to Amicus’ meds for Pompe and Fabry diseases, CEO Alexander Hardy is forging a key part of the future for BioMarin as part of a multiyear turnaround. The drugmaker has conducted layoffs , reorganizations and pipeline culls over the past few years to adjust the ship. “When I came to BioMarin two years ago, I came because I saw the potential for a really iconic biotech company and to really continue to strengthen our leadership position in rare diseases,” Hardy told Endpoints News . He said it quickly became apparent that the company had the global footprint and manufacturing capability “to produce all different types of modalities of drugs and reach patients around the world.” Hardy and chief business officer James Sabry have been working for more than a year to find a new direction for BioMarin as it looks to divest the hemophilia gene therapy marketed as Roctavian and grow its existing portfolio of medicines, including Palynziq and Voxzogo. While Sabry told Endpoints in January that they’re hunting for deals of a smaller size, mainly below $1.5 billion, Friday’s news suggests BioMarin needed a bigger deal with approved medicines to speed up its transformation. Amicus’ treatments have reached more than 3,400 patients globally, CEO Bradley Campbell said in a press release. BioMarin agreed to pay $14.50 a share, a 33% premium to Amicus’ closing price on Thursday, with the all-cash deal expected to close in the second quarter. Amicus’ share price increased 30% $FOLD , and BioMarin’s stock $BMRN climbed 19% in Friday trading. In conjunction with the deal announcement, BioMarin said Amicus inked settlement agreements with Aurobindo and Lupin over litigation related to Galafold. As part of the agreements, those generic manufacturers can’t enter the US market before Jan. 30, 2037. Amicus previously resolved a patent dispute with Teva in 2024. Hardy told Endpoints that BioMarin had approached Amicus about an acquisition, but wouldn’t complete the deal until Amicus resolved the legal disputes. “It was very important for us, we watched them, that the IP settlement would be concluded before any transaction happened,” Hardy said. Princeton, NJ-based Amicus was founded 23 years ago by John Crowley, who stepped away from the biotech a couple years ago to become CEO of Biotechnology Innovation Organization, the industry’s main advocacy group. Amicus has been on the market for multiple years. Galafold was approved in Europe in 2016 and in the US in 2018 for Fabry disease. Pombiliti + Opfolda was approved for Pompe disease in 2023 in both the US and Europe. Cantor Fitzgerald analyst Olivia Brayer wrote in a note to clients that the deal “makes a lot of sense,” even though it was “on the bigger side of what we’d been expecting” BioMarin to do. “It allows them to leverage their existing rare disease / enzyme therapy segment, and it immediately adds to BioMarin’s top-line growth – which has been a major focus for investors,” Brayer wrote. With the Amicus acquisition and the $270 million deal for Inozyme earlier this year now lined up, BioMarin expects its business development team to stay busy. “We’ll be looking at M&A, we’ll be looking at product partnerships, product acquisitions — all of the above, essentially,” Hardy said in the interview. “We need to use the cash-flow generation that we’ve created over the last couple of years, in addition to this deal, to regenerate our firepower. We believe we can do that within two years.” BioMarin had $2 billion in cash and investments at the end of the third quarter. It will use some of that available capital and $3.7 billion of non-convertible debt financing to buy Amicus. BioMarin recently guided to $3.2 billion in full-year revenues in 2025.

iStock, Sundry Photography 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.