Amicus Therapeutics

Amicus Therapeutics, Inc.

Type| Public

Traded as

| NASDAQ: FOLD
Russell 2000 component
Industry| Biotechnology
Founded| February 4, 2002; 24 years ago (2002-02-04)[1]: 28
Headquarters|

Philadelphia, Pennsylvania

United States

Key people

* John Crowley (chairman emeritus)
* Bradley Campbell (president and COO)
* Daphne Quimi (CFO)
* David Clark (chief people officer)

Revenue|

* $91,245,000 (2018)
* $36,930,000 (2017)

Operating income

* ($328,777,000) (2018)
* ($441,985,000) (2017)

Net income

* ($349,089,000) (2018)
* ($449,121,000) (2017)

Total assets|

* $789,951,000 (2018)
* $627,024,000 (2017)

Total equity|

* $342,912,000 (2018)
* $352,850,000 (2017)

Number of employees

| 508 (31 Dec 2018)[1]: 28
Website| www.amicusrx.com
Footnotes / references
[1][2]

Amicus Therapeutics, Inc. is a public American biopharmaceutical company based in Philadelphia, Pennsylvania.[3] The company went public in 2007 under the Nasdaq trading symbol FOLD.[4] This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX[5][6] Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates.[7]

## History

The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders.[8]: 2 The company has focused on pharmacological chaperones and enzyme replacement therapy.[8]: 2, 18 [9]: 95

In 2008, the company expanded from its single site in New Jersey to a second research site in San Diego.[10]

In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with Shire for its lead compound, migalastat, and two other products.[11]: 24 Amicus cut 20% of its workforce.[11]: 24

The next year, the company signed an exclusive license agreement with GlaxoSmithKline for migalastat; GSK paid $60 million in upfront and equity payments while promising up to $170 million to help develop their products,[12] GSK terminated the arrangement in 2013.[12]

In November 2013, Amicus acquired competitor Callidus Biopharma for its enzyme replacement therapy treatment for Pompe disease.[8]: 3, 12

In September 2015, the company acquired Scioderm for $229 million in cash and stock, with potentially $618 million more in biodollars. Scioderm had a drug in Phase III trials for epidermolysis bullosa; additionally, if the drug was approved the FDA would give its owner a priority review voucher, which itself can be sold to another company.[13] That drug failed its Phase III trial in 2017 and Amicus abandoned it. It had paid out around half of the milestones to Scioderm's shareholders prior to closing down the program.[14]

Also in September 2015, Amicus announced that it would submit a new drug application (NDA) for accelerated approval of migalastat to the FDA by the end of 2015.[15] An FDA committee recommended approval in April 2016, but the FDA rejected the application in November for having insufficient data in November 2016.[16] The drug was approved in the European Union in May 2016.[17]

In 2016 Amicus acquired MiaMed which was working on drugs to treat CDKL5 deficiency; Amicus paid $1.8M in cash and around $4.7M in stock, with $83M in biodollars.[18]

After Scott Gottlieb became FDA commissioner in 2017, the CEO of Amicus began lobbying him directly for the FDA to accept the NDA and in February 2018 the FDA accepted it and promised a response by August 2018.[19]

In September 2018, Amicus announced that it had acquired Celenex, which had ten early stage gene therapies. Amicus paid $100 million upfront and could pay an additional $352 million if developmental milestones are achieved.[20]

In December 2025, BioMarin Pharmaceutical agreed to acquire Amicus for $4.8 billion.[21]

## References

1. ↑ 1.0 1.1 1.2 "Form 10-K: Amicus Therapeutics, Inc.". United States Securities and Exchange Commission. 2018. p. 61. https://www.sec.gov/Archives/edgar/data/1178879/000117887919000024/amicus1231201810-k.htm.
2. ↑ "Amicus Leader, BIO Board Member John Crowley Named Trade Group’s New CEO". MedCity News. Dec 5, 2023. https://medcitynews.com/2023/12/bio-john-crowley-biotechnology-innovation-amicus-therapeutics/.
3. ↑ "Amicus Corporate Headquarters". https://www.amicusrx.com/about-us/contact-us.
4. ↑ "Amicus Therapeutics IPO raises $75 mln, in range". Reuters. 30 May 2007. https://www.reuters.com/article/amicustherapeutics-ipo-idUSWEN848220070531.
5. ↑ Gelsi, Steve (17 May 2006). "Amicus Therapeutics files $86M IPO". MarketWatch. http://www.marketwatch.com/story/amicus-therapeutics-files-86m-ipo.
6. ↑ Moyer, Liz (16 Aug 2006). "IPOs Head For The Exits". Forbes. https://www.forbes.com/2006/08/15/ipo-offerings-pulled-cx_lm_0816ipo.html. "There have been five withdrawn IPOs so far this month, including ... Amicus Therapeutics."
7. ↑ Rubin, Evelyn (18 May 2006). "Amicus IPO: A Very Risky Prospect (AMTX)". Seeking Alpha. http://seekingalpha.com/article/10870-amicus-ipo-a-very-risky-prospect-amtx.
8. ↑ 8.0 8.1 8.2 "Form 10-K: Amicus Therapeutics, Inc.". United States Securities and Exchange Commission. 2014. https://www.sec.gov/Archives/edgar/data/1178879/000104746914001702/a2218555z10-k.htm.
9. ↑ Williams, Cory (2014). "Chapter 4. Treating Rare Diseases: Business Model for Orphan Drug Development". in Pryde, David C.. Orphan Drugs and Rare Diseases. Royal Society of Chemistry. ISBN 9781849738064.
10. ↑ Staff (1 Oct 2008). "Amicus Therapeutics Opens Research Facility in San Diego.". Biotech Week (United States). http://www.highbeam.com/doc/1G1-185777890.html.
11. ↑ 11.0 11.1 "Form 10-Q: Amicus Therapeutics, Inc.". USSEC. 30 Sep 2009. https://www.sec.gov/Archives/edgar/data/1178879/000095012309057623/c91935e10vq.htm.
12. ↑ 12.0 12.1 Carroll, John (20 Nov 2013). "GlaxoSmithKline politely dumps $230M Fabry drug deal with Amicus". FierceBiotech. http://www.fiercebiotech.com/story/glaxosmithkline-politely-dumps-230m-fabry-drug-deal-amicus/2013-11-20.
13. ↑ Fidler, Ben (31 August 2015). "Xconomy: With $229M Deal, Amicus Gets a New Drug And Perhaps a Voucher". Xconomy. https://www.xconomy.com/new-york/2015/08/31/with-229m-deal-amicus-gets-a-new-drug-and-maybe-a-voucher/?single_page=true.
14. ↑ Taylor, Phil (September 13, 2017). "Amicus ditches skin disorder drug after late-stage failure" (in en). FierceBiotech. https://www.fiercebiotech.com/amicus-ditches-skin-disorder-drug-after-late-stage-failure.
15. ↑ "Amicus Therapeutics Plans to Submit NDA for Migalastat for Fabry Disease Following Positive Pre-NDA Meeting With FDA". Drugs.com. 15 September 2015. https://www.drugs.com/nda/migalastat_150915.html.
16. ↑ Adams, Ben (November 29, 2016). "FDA rejects quick Amicus Fabry drug read-through, new data not expected till 2019" (in en). FierceBiotech. https://www.fiercebiotech.com/biotech/fda-rejects-quick-amicus-fabry-drug-read-through-new-data-not-expected-til-2019.
17. ↑ "Amicus Therapeutics Announces European Commission Approval for Galafold (Migalastat) in Patients with Fabry Disease in European Union". GlobeNewswire. 31 May 2016. https://globenewswire.com/news-release/2016/05/31/844646/0/en/Amicus-Therapeutics-Announces-European-Commission-Approval-for-Galafold-Migalastat-in-Patients-with-Fabry-Disease-in-European-Union.html.
18. ↑ Adams, Ben (July 7, 2016). "Amicus spends $90M on MiaMed; gains rare disease preclinical program" (in en). FierceBiotech. https://www.fiercebiotech.com/biotech/amicus-spends-90m-miamed-gains-rare-disease-preclinical-program.
19. ↑ Carroll, John (February 12, 2018). "Once rejected, FDA now rolls out a short red carpet for Amicus' migalastat". Endpoints. https://endpts.com/once-rejected-fda-now-rolls-out-a-short-red-carpet-for-amicus-migalastat/.
20. ↑ Herper, Matthew. "A $100 Million Biotech Deal Is Also A Tale Of Two Executives Facing Their Kids' Deadly Diseases" (in en). Forbes. https://www.forbes.com/sites/matthewherper/2018/09/20/a-100-million-biotech-deal-is-also-a-tale-of-two-executives-facing-their-kids-deadly-diseases/#6ee26df82aad.
21. ↑ Kellaher, Colin; Glickman, Ben (2025-12-19). "BioMarin to Buy Rare-Disease Biotech Amicus Therapeutics for $4.8 Billion" (in en-US). The Wall Street Journal. https://www.wsj.com/business/deals/biomarin-to-buy-amicus-therapeutics-for-4-8-billion-c338bf7d.

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