INZ-701

BioMarin Pharmaceutical has entered into a definitive agreement to acquire rare disease biotech, Amicus Therapeutics for $4.8bn. Image credit: T. Schneider via ShutterStock.com. BioMarin Pharmaceutical has agreed to buy rare disease biotech Amicus Therapeutics, adding three approved or late-stage lysosomal storage disorder therapies to its portfolio. Under the terms of the agreement, BioMarin will pay cash for all of Amicus’ shares for a total consideration of $4.8bn. This deal will set BioMarin back $14.50 per share, constituting a 33% premium on Amicus’ $10.89 18 December stock value at market close. BioMarin’s stock has increased 17.71% on the acquisition, from an 18 December close of $51.95 to a 19 December close of $61.15. This multi-billion-dollar deal, which is the biggest in BioMarin’s history, will give the pharma rights to Amicus’ Fabry disease therapy, Galafold (migalastat), which has been on the US market since 2018. In 2024, the drug brought in $458.1m for Amicus, an 18% increase on 2023. Galafold is currently the only US Food and Drug Administration (FDA) approved chaperone therapy for Fabry disease – a rare genetic disorder characterised by the buildup of fatty substrates in the cells of an affected patient. Following a US patent litigation with generics giant Teva Pharmaceuticals, Galafold now holds market exclusivity until January 30, 2027. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence BioMarin will also acquire the rights to Amicus’ FDA-approved Pompe disease combination, Pombiliti (cipaglucosidase alfa-atga) plus Opfolda (miglustat), which got the FDA go-ahead for use in patients who are not responding to standard enzyme replacement therapy back in 2023. The drug generated $22m in 2024. Like Fabre disease, Pompe disease is a lysosomal storage disorder that prevents the metabolism of glycogen, leading to its harmful buildup within the cells. This can lead to progressive muscular weakness and heart problems. Alongside these approved therapies, BioMarin will take ownership of the US rights to the late-stage, investigational oral small molecule, DMX-200, which is currently in Phase III trials for rare kidney disease, focal segmental glomerulosclerosis (FSCS). Amicus originally purchased the US rights to DMX-200 from the drug’s creator, Dimerix, for up to $105m in May 2025. BioMarin eyes future growth Over the last couple of years, BioMarin has been looking to further its growth in the pharmaceutical sector. In a bid to achieve this, the company underwent a significant restructuring in 2024, which saw it let go of 225 employees. Moving into 2025, BioMarin has employed a strong dealmaking strategy, which saw the company sign 10 deals in 2025. According to GlobalData’s Pharmaceutical Intelligence Center, this is the highest number of deals to be signed in one year since 2010. One of the more prominent deals was BioMarin’s acquisition of Inozyme , which set it back $270m, while adding Inozyme’s late-stage enzyme replacement therapy, INZ-701, to its pipeline. Following its merger with Amicus, BioMarin noted that both Galafold and Pombiliti-Opfolda have “high growth potential,” meaning the acquisition could further accelerate the company’s revenue growth moving forward. This could be further supported by the expansion of Galafold and Pombiliit-Opfolda’s global footprint, which BioMarin mentioned as a possibility in a 19 December statement. Analysts at GlobalData, parent company of Pharmaceutical Technology , currently forecast that Galafold will generate sales of $840m in 2031, while Pombiliti-Opfolda is expected to bring in $691m during the same year. Pharmaceutical Technology Excellence Awards - Nominations Closed Nominations are now closed for the Pharmaceutical Technology Excellence Awards . A big thanks to all the organisations that entered – your response has been outstanding, showcasing exceptional innovation, leadership, and impact. Excellence in Action Awarded the 2025 Pharmaceutical Technology Excellence Award for Business Expansion in Integrated Manufacturing , Upperton Pharma Solutions is rapidly expanding its UK GMP and sterile manufacturing footprint. Find out how Upperton’s integrated CDMO model helps pharma companies move from early development to clinical and niche commercial supply with fewer handovers and faster timelines. Discover the Impact

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.