Pharma BD Deal Intelligence

BioMarin Pharmaceutical Inc. / Amicus Therapeutics, Inc.

2025 · Acquisition/Merger · $4.8B · Complete

BioMarin's largest-ever deal: $4.8B cash, a 33% premium, for Amicus's Galafold and Pombiliti+Opfolda—commercial rare-disease revenue now, not pipeline promises—though it required $3.7B in new debt to finance.

Outcome grade pending — assessed 5 years post-close.

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The coverage arc

Dec 19, 2025 Amicus Therapeutics 8-K Filing Neutral

On December 19, 2025, Amicus Therapeutics, Inc. entered into an Agreement and Plan of Merger with BioMarin Pharmaceutical Inc. and a wholly-owned subsidiary,…

Dec 19, 2025 MedCity News Bullish

BioMarin's Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition. Leerink Partners analyst Joseph Schwartz: the acquisition of…

Dec 22, 2025 Fierce Biotech Bearish

BioMarin quietly discards liver disease candidate after $4.8B Amicus announcement. Three days after announcing its largest-ever acquisition, BioMarin disclosed…

Source summaries from our enrichment pipeline; follow links for originals.

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BioMarin completed its acquisition of Amicus Therapeutics on April 27, 2026, for $14.50/share in cash, total equity value ~$4.8B (a 33% premium) - BioMarin's largest-ever transaction. The deal closed as a one-step all-cash merger after Amicus stockholders approved it and the final regulatory condition (France Ministry of Economics and Finance clearance, April 23, 2026) was satisfied; U.S. FTC granted HSR early termination on February 11, 2026. Amicus survives as a wholly owned BioMarin subsidiary and was delisted from Nasdaq. The acquisition strengthens BioMarin's rare disease portfolio with two commercial lysosomal storage disorder therapies - Galafold (migalastat) for Fabry disease and Pombiliti+Opfolda (cipaglucosidase alfa-atga + miglustat) for Pompe disease - plus the late-stage kidney asset DMX-200 (Phase 3, FSGS).

Key facts

Disease & market context

Fabry Disease, Pompe Disease, and FSGS (Rare Disease Portfolio)

50K US cases/yr · $4.5B Combined US Rare Disease Franchise Market

Disease Overview

The acquisition consolidates three distinct rare disease indications. Fabry disease is an X-linked lysosomal storage disorder caused by GLA gene mutations leading to alpha-galactosidase A deficiency and progressive accumulation of globotriaosylceramide (Gb3), resulting in renal failure, cardiomyopathy, stroke, and neuropathic pain; approximately 3,000-10,000 US patients. Pompe disease (glycogen storage disease type II) is caused by GAA gene mutations producing acid alpha-glucosidase deficiency, leading to glycogen accumulation in muscle and cardiac tissue with infantile-onset and late-onset phenotypes; approximately 3,000-5,000 US patients. Focal segmental glomerulosclerosis (FSGS) is a histologic pattern of kidney injury characterized by scarring in parts of some glomeruli, causing nephrotic syndrome, progressive chronic kidney disease, and end-stage renal disease; approximately 40,000 US patients with primary FSGS, no FDA-approved disease-modifying therapies currently. Standard of care across these indications ranges from enzyme replacement therapy (ERT), chaperone therapy, substrate reduction therapy, and symptomatic management; FSGS is limited to immunosuppression and RAAS blockade.

Competitive Landscape

The rare disease competitive landscape spans multiple mechanisms. In Fabry disease: Fabrazyme (agalsidase beta, Sanofi) as enzyme replacement therapy, Replagal (agalsidase alfa, Takeda, ex-US), Galafold (migalastat, Amicus) as oral pharmacological chaperone for amenable GLA mutations, and next-generation approaches including Sanofi's venglustat (substrate reduction) and Protalix/Chiesi's pegunigalsidase alfa (Elfabrio). In Pompe disease: Myozyme/Lumizyme (alglucosidase alfa, Sanofi) and Nexviazyme (avalglucosidase alfa, Sanofi) as enzyme replacement, Pombiliti+Opfolda (cipaglucosidase alfa + miglustat, Amicus) as ERT plus stabilizer, and AAV-based gene therapies from Sarepta, Spark, and Astellas. In FSGS: no approved disease-modifying therapy; Travere's sparsentan (Filspari, dual ETA/ARB) approved for IgAN with FSGS Phase 3 DUPLEX; DMX-200 (Dimerix/Amicus, CCR2 antagonist); Vertex's inaxaplin (APOL1-mediated); Goldfinch's GFB-887; Variant Bio earlier-stage programs.

Deal timeline

Related deals — scored

DealYearValueOutcome
BioMarin Pharmaceutical Inc. / Amicus Therapeutics, Inc. (this deal)2025$4.8B
BioMarin Pharmaceutical Inc. / Inozyme Pharma, Inc.2025$270M
BioMarin Pharmaceutical Inc. / Alesta Therapeutics B.V.2026$490M
Shire plc / Dyax Corp.2016$5.9B89
Horizon Pharma plc / Hyperion Therapeutics, Inc.2015$1.1B79
Shire plc / NPS Pharmaceuticals2015$5.2B78
AstraZeneca PLC / Amolyt Pharma2024$1.1B73

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