Cars Changed the World Once—Now They’re About to Change It Again
BioMarin to acquire Amicus Therapeutics for $4.8 billion in rare disease bet
BioMarin Pharmaceutical has agreed to acquire Amicus Therapeutics for $4.8 billion in an all-cash deal, marking its largest transaction ever and bolstering its leadership in rare disease treatments. The acquisition adds two approved therapies—Galafold for Fabry disease and Pombiliti/Opfolda for Pompe disease—to BioMarin's portfolio, alongside a late-stage kidney disease candidate. This strategic move aims to accelerate revenue growth through 2030 by diversifying into high-value lysosomal storage disorders.
Deal Details
BioMarin will pay $14.50 per Amicus share, a 33% premium to the prior closing price, 46% over the 30-day average, and 58% above the 60-day average, valuing the equity at roughly $4.8 billion. Financing combines BioMarin's cash reserves with $3.7 billion in non-convertible debt arranged by Morgan Stanley. Both boards unanimously approved the pact, with closure targeted for Q2 2026 pending shareholder votes, regulatory nods, and standard conditions.
Strategic Fit and Assets
BioMarin CEO Alexander Hardy called Amicus an "exceptional strategic fit," citing its global reach, manufacturing prowess, and enzyme therapy synergies. Galafold, an oral chaperone for Fabry (affecting 1 in 40,000-117,000 births), posted $371 million in nine-month 2025 sales, with patents now secured against generics until 2037 via recent settlements. Pombiliti (enzyme replacement) plus Opfolda (chaperone) targets Pompe, a muscle-wasting disorder, while DMX-200 advances in phase 3 for focal segmental glomerulosclerosis (FSGS), a rare kidney condition.
Financial Impact
The deal projects accretion to non-GAAP EPS within 12 months post-close, folding Amicus into BioMarin's enzyme unit that drove 11% year-over-year growth to $2.3 billion in nine-month revenue. Analysts praise the diversification bet, though Stifel notes risks in peak sales assumptions for the assets. BioMarin's stock surged 19% to over $62 on announcement day, reflecting investor enthusiasm for expanded scale.
Market and Competitive Context
Both firms specialize in ultra-rare diseases, where single drugs generate blockbuster potential due to orphan drug exclusivity and pricing. BioMarin's move counters patent cliffs on flagships like Voxzogo, while Amicus gains BioMarin's resources for faster global launches. The transaction resolves Galafold litigation with Aurobindo and Lupin, extending U.S. monopoly and supporting long-term growth projections.
Leadership and Integration Plans
BioMarin executives emphasize seamless integration, retaining Amicus CEO John Crowley in an advisory role to leverage his rare disease advocacy expertise. The combined entity will centralize R&D in San Rafael, California, accelerating DMX-200's phase 3 readout expected mid-2026, potentially adding a third blockbuster. Manufacturing synergies cut costs by 15-20% through shared enzyme facilities, enhancing supply chain resilience amid global shortages.
Regulatory and Shareholder Pathways
Antitrust scrutiny remains low given non-overlapping portfolios—BioMarin focuses on skeletal dysplasias and hemophilia, Amicus on metabolic disorders—clearing FTC review swiftly. Amicus shareholders receive $14.50 cash per share, with tender offer alternatives if votes falter. Kirkland & Ellis advised Amicus, while BioMarin tapped Morgan Stanley for financing, signaling confidence in debt markets despite biotech volatility.
Broader Industry Trends
This $4.8 billion deal exemplifies 2025's M&A surge in rare diseases, where orphan drugs yield 20-30% margins versus 5-10% in oncology. Peers like PTC Therapeutics eye similar consolidations post-Sarepta partnerships. Analysts forecast the duo's pipeline peaking at $6-8 billion annual sales by 2030, driven by label expansions into pediatrics and emerging markets.
Patient and Advocacy Impact
Rare disease groups applaud the merger for bolstering access to Galafold, now reaching 10,000+ Fabry patients globally, and Pombiliti expanding to Asia. Global Genes highlights faster trials for FSGS, affecting 40,000 Americans, with orphan incentives intact. Critics note pricing pressures, but CEOs commit to patient assistance programs covering 90% of eligible U.S. cases.
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