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BioMarin has executed the largest acquisition in its 28-year history, gaining Amicus Therapeutics for $4.8 billion. With the move, BioMarin's share price increased by 18% Friday. Sixteen months after luring former Roche dealmaker James Sabry, M.D., Ph.D., out of retirement and signaling a shift in its business development approach, BioMarin has announced the largest transaction in the company’s 28-year history.In a merger of rare disease specialists, BioMarin has agreed to acquire Amicus Therapeutics for $4.8 billion. The California biopharma will pay $14.50 per share, which is a 33% premium on the $10.89 Thursday closing price of the New Jersey-based biopharma and a 46% premium on its 30-day average.With the deal, BioMarin gains two rapidly growing products—Fabry disease drug Galafold and Pompe disease combination treatment Pombiliti-Opfolda. BioMarin also acquires the U.S. rights to DMX-200, a potential first-in-class small molecule in phase 3 development for the rare kidney disease focal segmental glomerulosclerosis (FSGS).Galafold, which was approved in 2018, generated sales of $458 million in 2024 for an 18% year-over-year increase. The Pombiliti-Opfolda combo, approved in September of 2023, generated $70 million in sales last year. Through the first 9 months of this year, the combo's sales reached $78 million.“This deal will accelerate BioMarin’s revenue growth immediately upon close, adding two high-growth products with numerous global expansion opportunities,” CEO Alexander Hardy said on a conference call. “Galafold and (Pombiliti-Opfolda) each have the potential to reach $1 billion in peak sales.”With its portfolio of enzyme replacement therapies accounting for 68% of its revenue last year, BioMarin recorded sales of $2.85 billion, an increase of 18% year over year. This year, however, the sales momentum has slowed to an 11% increase over the first nine months and a 4% boost in the third quarter.Investors were bullish on the move as BioMarin’s share price increased by 18% by mid-morning on Friday.“This deal clearly is value-creating,” Hardy said. “It’s an exceptional strategic fit.”BioMarin chief commercial officer Cristin Hubbard noted that the company sees significant growth potential with Galafold because Fabry disease is “a dramatically underdiagnosed condition.”“It’s estimated today that there are 18,000 patients that have been diagnosed, but there are 6,000 that aren’t treated. That right there is a really big opportunity for us to think about where Galafold could play,” Hubbard said. As for Pompe disease, Hubbard said that BioMarin has a “unique opportunity to leverage our scale to accelerate the growth of these assets.” Hubbard noted that the combo is reimbursed in only 15 countries and there’s much room for expansion across BioMarin’s “nearly 80-country footprint.”BioMarin's growth potential is also tied to Voxzogo, which generated sales of $735 million last year and is the only drug on the market which treats a form of dwarfism in children called achondroplasia. The treatment is expected to soon face competition from Ascendis Pharma's TransCon CNP, which is scheduled for an FDA decision in the first quarter of next year.In a note to clients, Evercore ISI analyst Cory Kasimov wrote that his team views the deal “as a smart, strategic add-on that leverages BioMarin’s established rare disease commercialization capabilities while extending its enzyme replacement franchise” into the Fabry disease and Pompe disease markets. It is BioMarin’s second key acquisition of the year after the drugmaker gained Inozyme for $270 million. The May deal added enzyme replacement therapy INZ-701, which is in phase 3 development with topline data expected early next year.Throughout its history, BioMarin has largely been focused on early-stage deals. And in fact, more than a decade ago, it was seen as a takeover target before and after the 2014 approval of replacement therapy Vimizim, which is now the company’s top-selling product, generating $740 million last year.BioMarin expects the acquisition of Amicus to close in the second quarter of next year.

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.