BioMarin Pharmaceutical, Inc.

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.

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.

BioMarin Pharmaceutical agreed to buy Amicus Therapeutics for $14.50 per share in cash, or approximately $4.8 billion, adding treatments for Fabry and Pompe to its rare disease portfolio. The deal, announced Friday, represents a 33% premium to Amicus' closing share price on Dec. 18 and has been approved by the boards of both companies. "Immediately upon close, this transaction is expected to accelerate BioMarin's revenue growth and strengthen our financial outlook," remarked CEO Alexander Hardy. The takeout is slated to be finalised in the second quarter of 2026.Over the past four quarters, combined sales of Amicus' Fabry disease drug Galafold (migalastat) and the dual regimen of Pombiliti (cipaglucosidase alfa-atga) plus Opfolda (miglustat) for Pompe disease have reached $599 million. BioMarin noted that the products all have "high-growth potential" and are expected to boost its revenue through 2030 and beyond. "[Our] scale of operations, including our global commercial footprint and industry-leading, in-house manufacturing capabilities make the combination of these companies an exceptional strategic fit," Hardy said.Along with its approved drugs, Amicus also has US rights to DMX-200, an investigational small molecule in Phase III development for the treatment of focal segmental glomerulosclerosis. Galafold litigation settledIn conjunction with the transaction, Amicus has resolved US patent litigation related to generic versions of Galafold from Aurobindo Pharma and Lupin, delaying their entry until January 30, 2037. The company had previously reached a similar deal with Teva.Under the new settlements, the parties will terminate all ongoing litigation regarding Galafold patents pending in the District Court for the District of Delaware.