BioMarin's $4.8B Amicus Buy: A Power Play in Rare Disease

SAN RAFAEL, CA – December 19, 2025 – In a decisive move to solidify its dominance in the rare disease sector, BioMarin Pharmaceutical Inc. announced today it will acquire Amicus Therapeutics for approximately $4.8 billion. The all-cash transaction, priced at $14.50 per share, marks a significant consolidation in the biotechnology landscape and sent Amicus's stock soaring nearly 30% in premarket trading, signaling strong investor approval.

The acquisition, unanimously approved by both boards, will transfer two high-growth commercial therapies and a promising late-stage pipeline asset to BioMarin, immediately accelerating its revenue and strengthening its position as a leader in treating lysosomal storage disorders. The deal is expected to close in the second quarter of 2026, pending customary regulatory and stockholder approvals.

A Strategic Overhaul for Market Leadership

At the heart of the acquisition is a powerful strategic alignment. BioMarin will absorb two key Amicus products: Galafold® (migalastat), a first-in-class oral therapy for Fabry disease, and Pombiliti® + Opfolda®, a two-component treatment for late-onset Pompe disease. Together, these therapies have already demonstrated significant market traction, generating a combined $599 million in revenue over the last four quarters.

"Amicus, like BioMarin, is a company that has been profoundly dedicated to transforming care for patients with rare diseases since its founding," said Alexander Hardy, President and Chief Executive Officer of BioMarin, in a statement. He emphasized that BioMarin's scale, global commercial footprint, and in-house manufacturing capabilities make the combination an "exceptional strategic fit."

This move is a cornerstone of BioMarin's strategy under Hardy, who took the helm in late 2023 with a vision to reach $4 billion in annual revenue by 2027. The deal is projected to be immediately accretive to non-GAAP earnings per share within the first year and substantially so by 2027, providing a direct and powerful boost to the company's financial outlook. Critically, the acquisition also resolves pending patent litigation for Galafold, extending its U.S. market exclusivity through January 2037—a multi-billion dollar assurance that underpins the deal's long-term value.

Decoding the Premium Price Tag

The $4.8 billion valuation, which represents a 33% premium over Amicus's last closing price, reflects more than just its current revenue stream. It's a calculated investment in a diversified and growing portfolio within a lucrative market. The global market for Fabry disease treatment is projected to exceed $5 billion by 2032, while the Pompe disease market is expected to surpass $2.3 billion. Galafold's oral formulation offers a convenient alternative to traditional enzyme replacement therapies, and Pombiliti + Opfolda represents a next-generation option for patients.

Beyond these established products, the deal includes a significant pipeline asset: DMX-200. This investigational molecule is in Phase 3 trials for focal segmental glomerulosclerosis (FSGS), a rare and devastating kidney disease with no currently approved specific treatments. Success with DMX-200 would position BioMarin to enter a market with a profound unmet medical need, estimated to be worth over $20 billion by 2030.

To fund the acquisition, BioMarin will use cash on hand and approximately $3.7 billion in new debt. The company has laid out a clear deleveraging plan, targeting a gross leverage of less than 2.5 times within two years of the deal's closing, signaling a fiscally disciplined approach to this major expansion.

Beyond the Boardroom: The Patient Perspective

While the financial and strategic implications are clear, the merger's ultimate impact will be measured in the lives of patients. Leaders from both companies have framed the acquisition as a victory for the rare disease community.

"With BioMarin's unwavering commitment to patients, along with greater resources and scale, Amicus' medicines will reach even more patients around the world, faster," stated Bradley L. Campbell, President and Chief Executive Officer of Amicus. The promise is that BioMarin's extensive global infrastructure can expand access to Galafold and Pombiliti + Opfolda into new markets, bringing established treatments to underserved populations.

However, large-scale pharmaceutical mergers often prompt cautious observation from patient advocacy groups and healthcare providers. Concerns typically center on the potential for price increases, changes to patient support programs, and the continuity of research and development. The high cost of orphan drugs is a persistent challenge for the rare disease community, and any shifts in corporate strategy following the merger will be closely monitored. The future of Amicus's patient-centric culture and its pipeline, particularly the DMX-200 program for FSGS, will be a key area of focus for patient communities who depend on such innovation.

Navigating Integration and a Shifting Regulatory Tide

The path to realizing the full value of this acquisition is not without its challenges. BioMarin has a mixed track record with major acquisitions; while some have successfully bolstered its pipeline, others, like the 2014 purchase of Prosensa for its Duchenne muscular dystrophy program, ultimately did not yield an approved therapy. Successful integration will require a deft merging of corporate cultures, commercial teams, and research priorities.

The deal also comes at a time of heightened regulatory scrutiny of M&A in the pharmaceutical industry, with bodies like the U.S. Federal Trade Commission (FTC) taking a more aggressive stance. The transaction will require clearance under the Hart-Scott-Rodino Act and from other international antitrust authorities. However, the complementary nature of the two companies' portfolios may help smooth the path to approval.

With the transaction expected to close in the second quarter of 2026, the coming months will be critical. Amicus will seek approval from its stockholders, and both companies will navigate the complex regulatory landscape. For BioMarin, this acquisition represents a bold, transformative step, betting billions on a future where its leadership in the rare disease space is not just maintained, but decisively expanded.