Amicus Therapeutics is a public American

biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, t ...

company based in

Philadelphia, PA. Philadelphia, often called Philly, is the List of municipalities in Pennsylvania#Municipalities, largest city in the Commonwealth (U.S. state), Commonwealth of Pennsylvania, the List of United States cities by population, sixth-largest city i ...

The company went public in 2007 under the

NASDAQ The Nasdaq Stock Market () (National Association of Securities Dealers Automated Quotations Stock Market) is an American stock exchange based in New York City. It is the most active stock trading venue in the US by volume, and ranked second ...

trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of

venture capital Venture capital (often abbreviated as VC) is a form of private equity financing that is provided by venture capital firms or funds to startups, early-stage, and emerging companies that have been deemed to have high growth potential or which ha ...

firms including Radius Ventures, Canaan Partners and

New Enterprise Associates New Enterprise Associates (NEA) is an American-based venture capital firm. NEA focuses investment stages ranging from seed stage through growth stage across an array of industry sectors. With ~$25 billion in committed capital, NEA is one of the w ...

History

The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called

lysosomal storage disorders Lysosomal storage diseases (LSDs; ) are a group of over 70 rare inherited metabolic disorders that result from defects in lysosomal function. Lysosomes are sacs of enzymes within cells that digest large molecules and pass the fragments on to other ...

. The company has focused on

pharmacological chaperone A pharmacological chaperone or pharmacoperone is a drug that acts as a protein chaperone. That is, it contains small molecules that enter cells and serve as a molecular scaffolding in order to cause otherwise- misfolded mutant proteins to fold an ...

s and

enzyme replacement therapy Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is availab ...

. In 2008, the company expanded from its single site in New Jersey to a second research site in

San Diego San Diego ( , ; ) is a city on the Pacific Ocean coast of Southern California located immediately adjacent to the Mexico–United States border. With a 2020 population of 1,386,932, it is the List of United States cities by population, eigh ...

. In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with

Shire Shire is a traditional term for an administrative division of land in Great Britain and some other English-speaking countries such as Australia and New Zealand. It is generally synonymous with county. It was first used in Wessex from the beginn ...

for its lead compound,

migalastat Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004 ...

, and two other products. Amicus cut 20% of its workforce. The next year, the company signed an exclusive license agreement with

GlaxoSmithKline GSK plc, formerly GlaxoSmithKline plc, is a British multinational pharmaceutical and biotechnology company with global headquarters in London, England. Established in 2000 by a merger of Glaxo Wellcome and SmithKline Beecham. GSK is the ten ...

for migalastat; GSK paid $60 million in upfront and equity payments while promising up to $170 million to help develop their products, GSK terminated the arrangement in 2013. In November 2013, Amicus acquired competitor Callidus Biopharma for its enzyme replacement therapy treatment for Pompe disease. 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 Epidermolysis bullosa (EB) is a group of rare medical conditions that result in easy blistering of the skin and mucous membranes. Blisters occur with minor trauma or friction and are painful. Its severity can range from mild to fatal. Inherited E ...

; additionally, if the drug was approved the FDA would give its owner a

priority review voucher Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

, which itself can be sold to another company. 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. Also in September 2015, Amicus announced that it would submit a new drug application (NDA) for

accelerated approval The United States Food and Drug Administration (FDA) initiated the FDA Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need. The faster approval relies on use of surrogate endp ...

to the FDA by the end of 2015. An FDA committee recommended approval in April 2016, but the FDA rejected the application in November for having insufficient data in November 2016. The drug was approved in the European Union in May 2016. In 2016 Amicus acquired MiaMed which was working on drugs to treat

CDKL5 CDKL5 is a gene that provides instructions for making a protein called cyclin-dependent kinase-like 5 also known as serine/threonine kinase 9 (STK9) that is essential for normal brain development. Mutations in the gene can cause deficiencies in t ...

deficiency; Amicus paid $1.8M in cash and around $4.7M in stock, with $83M in biodollars. After

Scott Gottlieb Scott Gottlieb (born June 11, 1972) is an American physician and investor who served as the 23rd commissioner of the Food and Drug Administration (FDA) from May 2017 until April 2019. He is presently a senior fellow at the conservative think tan ...

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. 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.

References

{{reflist 2002 establishments in New Jersey Drug discovery companies Pharmaceutical companies based in New Jersey Orphan drug companies Gene therapy Biotechnology companies of the United States Companies listed on the Nasdaq