Amicus Therapeutics, Inc. operates as a biotechnology company. The firm focuses on discovering, developing and delivering medicines for people living with metabolic diseases. Its product portfolio includes first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants, a novel, clinical-stage, treatment paradigm for Pompe disease and a rare disease gene therapy portfolio. The company was founded by John Francis Crowley on February 4, 2002 and is headquartered in Princeton, NJ.
Clinical Trials
121
72 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2002
Active, not recruiting
70
57.9%
Available
3
2.5%
Completed
32
26.4%
Not yet recruiting
2
1.6%
Recruiting
8
6.6%
Terminated
6
5.0%
No approval data available
- BioMarin Pharmaceutical will acquire privately held Alesta Therapeutics for $275 million up front, with up to $215 million more in development and regulatory milestones. - Alesta's lead candidate ALE1 is in a Phase 1/2 trial for hypophosphatasia, a genetic disease affecting bone mineralization that can cause fractures, tooth loss, and muscle weakness. - ALE1 acts on a novel target that lowers inorganic pyrophosphate, a metabolite central to hypophosphatasia, and could reach BioMarin's largest addressable patient population. - The deal, BioMarin's third acquisition in two years, is expected to close by the end of September and will be funded with cash on hand.
- BioMarin has completed its acquisition of Amicus Therapeutics at the end of April, integrating Fabry and Pompe disease communities into its rare disease portfolio. - Chief Commercial Officer Cristin Hubbard emphasized the company's goal to maintain momentum while leveraging BioMarin's global footprint across approximately 80 countries to expand patient access. - The company plans to apply its expertise in genomics, diagnostics, and AI-driven patient identification to help reach more people living with these rare genetic conditions. - The integration aims to deliver therapies without disruption while adding BioMarin's specialized capabilities in identifying underdiagnosed and misdiagnosed patient populations worldwide.
- Sangamo Therapeutics' gene therapy ST-920 (isaralgagene civaparvovec) is advancing toward accelerated FDA approval with a BLA submission planned for the latter half of 2025, following positive Phase 1/2 STAAR trial data. - The global Fabry disease treatment market was valued at USD 2.62 billion in 2025 and is projected to reach USD 5.92 billion by 2035, growing at a CAGR of 8.47%. - Enzyme replacement therapy remains the standard of care with 76.10% market share in 2025, while gene therapies and oral chaperone treatments are expected to reshape the treatment landscape. - In 2024, approximately 9,200 diagnosed prevalent cases of Fabry disease were estimated in the United States, representing 52% of the total across the seven major markets.
- BioMarin Pharmaceutical completed its first-ever senior notes offering, raising $850 million through 5.500% senior notes due 2034 to support strategic expansion. - The proceeds will finance BioMarin's acquisition of Amicus Therapeutics and debt repayment, combining two rare disease-focused biotechnology companies. - This transaction represents a significant consolidation move in the rare disease therapeutics sector, bringing together over 25 years of BioMarin's pioneering experience with Amicus's complementary portfolio.
- BioMarin Pharmaceutical announced the pricing of $850 million in 5.500% senior unsecured notes due 2034 to help finance its pending acquisition of Amicus Therapeutics. - The company completed syndication of a new $2 billion senior secured term loan facility, bringing total new debt facilities to $3.4 billion including revolving credit. - Net proceeds from the notes offering will be held in escrow until the Amicus acquisition closes, with mandatory redemption required if the deal fails to complete by December 19, 2026. - The transaction represents a significant expansion for BioMarin's rare disease portfolio, combining resources from two established biotechnology companies focused on genetic conditions.
- BioMarin Pharmaceutical announced the acquisition of Amicus Therapeutics for $4.8 billion in an all-cash transaction valued at $14.50 per share, representing a 33% premium to Amicus' last close. - The acquisition adds two commercial therapies to BioMarin's portfolio: Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease, which generated combined revenues of $599 million over the past four quarters. - The transaction is expected to be accretive to Non-GAAP Diluted EPS in the first 12 months after close and substantially accretive beginning in 2027, while strengthening BioMarin's position in the rare disease market. - Amicus resolved patent litigation for Galafold, securing U.S. exclusivity through January 2037 following settlement agreements with generic manufacturers Aurobindo Pharma and Lupin Ltd.
- Dimerix has successfully completed recruitment of 286 adult patients for its ACTION3 Phase 3 trial evaluating DMX-200 in focal segmental glomerulosclerosis (FSGS), a rare kidney disease. - The trial demonstrated positive interim results in March 2024, showing DMX-200 outperformed placebo in reducing proteinuria, a key marker of kidney disease progression. - With 94% of completed patients entering the open-label extension study and seven successful safety reviews, the company is positioned to advance toward regulatory submission with partner Amicus Therapeutics.
- Galafold (migalastat) generated $371.5 million in sales during the first nine months of 2025, representing a 12% year-over-year increase and contributing over 80% of Amicus Therapeutics' net product revenues. - The company secured patent protection for Galafold through 2038 and reached a settlement with Teva Pharmaceuticals preventing generic competition until January 2037. - Amicus' combination therapy Pombiliti + Opfolda for late-onset Pompe disease showed robust growth with $77.5 million in sales, up 61% year-over-year. - BioMarin has agreed to acquire Amicus Therapeutics for $4.8 billion, with the transaction expected to close in the second quarter of 2026.
- A comprehensive pipeline analysis reveals 12+ companies developing 15+ novel therapies for focal segmental glomerulosclerosis (FSGS), a rare kidney disorder with limited treatment options. - Travere Therapeutics' sparsentan (Filspari) has received FDA acceptance for supplemental approval following successful DUPLEX trial results showing sustained proteinuria reduction in FSGS patients. - Survey data from 102 U.S. nephrologists indicates only half of FSGS patients are considered optimally managed, driving strong physician interest in emerging therapies targeting APOL1-mediated disease. - The next 12-18 months are expected to redefine FSGS treatment standards, with multiple agents advancing toward regulatory milestones and potential FDA decisions in early 2026.
- Japan's Ministry of Health, Labour and Welfare has approved Pombiliti + Opfolda for treating adult patients with late-onset Pompe disease, expanding global access to this innovative therapy. - The approval was based on data from the Phase 3 PROPEL study, which uniquely studied both treatment-naïve and treatment-experienced patients in a controlled setting. - Pombiliti + Opfolda is now approved in seven major markets including the US, EU, UK, Canada, Australia, Switzerland, and Japan. - The two-component therapy combines a recombinant human GAA enzyme with enhanced muscle cell uptake and an oral enzyme stabilizer designed to maintain enzyme activity in blood.
