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Amicus Therapeutics

Amicus Therapeutics

A biotechnology company developing therapies to cure rare diseases

Amicus Therapeutics is a publicly listed biotechnology company focused on the discovery, development, and delivery of treatments for rare metabolic diseases. Headquartered in New Jersey, the company also operates officers internationally. They include the UK, Canada, Japan, Germany, France, Italy, The Netherlands, Spain, and Australia.

Products
Medication for Fabry disease

Amicus' developments include one commercialized medication for Fabry disease, an investigational enzyme replacement therapy (AT-GAA) in late stage development for the treatment of Pompe disease, and several gene therapy pipeline and growth platform for lysosomal storage disorders, including Batten Disease, Neimann-Pick Type C, Wolman Disease, and Tay Sachs.

In August 2018, the US FDA approved Amicus Therapeutics' Galafold, the first oral therapy to treat Fabry disease, a rare and sometime fatal condition in which accumulation of fat damages several organs. It is a long-term treatment for adult patients with specific genetic mutations, which account for an estimated 35 to 50% of diagnosed cases globally. Chemically known as migalastat, the drug has also been approved in Europe, Japan, Australia, and Canada.

Medication for Pompe disease

The company's investigational therapy, AT-GAA, for Pompe disease, an inherited Lysosomal Storage (LSD) caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), completed a Phase 2 safety and pharmacokinetics study in 2013.

Gene Therapy for Batten disease

Amicus is also conducting multiple Phase 1/2 studies for their CLN6 gene therapy to treat Batten disease, a broad class of rare, fatal, inherited disorders of the nervous system known as neuronal ceroid lipofuscinoses (NCLs).

Funding
Series A

On July 25, 2002 Amicus Therapeutics completed their series A funding round with $2 million in funding from CHL Medical Partners.

Series B

On May 13, 2004 Amicus Therapeutics completed their series B funding round with $31 million in funding from Canaan Partners (lead investor), Radius Venture Partners, New Enterprise Associates, Frazier Healthcare Partners, and CHL Medical Partners.

Series C

On September 8, 2005 Amicus Therapeutics completed their series C funding round with $55 million in funding from Quaker BioVentures (lead investor), Radius Ventures, Prospect Venture Partners, Palo Alto Investors, New Enterprise Associates, Garden State Life Sciences Venture, Frazier Healthcare Partners, Canaan Partners, and CHL Medical Partners.

Timeline

March 30, 2019
Series B funding round

On May 13, 2004 Amicus Therapeutics completed their series B funding round with $31 million in funding from Canaan Partners (lead investor), Radius Venture Partners, New Enterprise Associates, Frazier Healthcare Partners, and CHL Medical Partners.

March 30, 2019
Series C funding round

On September 8, 2005 Amicus Therapeutics completed their series C funding round with $55 million in funding from Quaker BioVentures (lead investor), Radius Ventures, Prospect Venture Partners, Palo Alto Investors, New Enterprise Associates, Garden State Life Sciences Venture, Frazier Healthcare Partners, Canaan Partners, and CHL Medical Partners.

July 25, 2002
Series A funding round

On July 25, 2002 Amicus Therapeutics completed their series A funding round with $2 million in funding from CHL Medical Partners.

February 4, 2002
Amicus Therapeutics was founded by John Crowley.

Funding rounds

Patents

Products

People

Name
Role
LinkedIn

Adriane Schilling

Employee

Allan Powe

Employee

Anne-Lise Wohlers

Employee

Bradley L. Campbell

President & COO

Christopher Holterhoff

Employee

...

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Companies

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News

Title
Author
Date
Publisher
Description
Alex Keown
July 2, 2021
BioSpace
Biopharma and life sciences companies strengthen their leadership teams and boards with these Movers & Shakers.
BioSpace
June 29, 2021
BioSpace
Amylyx Pharmaceuticals, Inc. today announced the addition of Daphne Quimi to its Board of Directors. Ms. Quimi brings more than 25 years of executive experience in the pharmaceutical and biotechnology industries with expertise in global finance operations, company building, and rare disease drug commercialization.
BioSpace
June 14, 2021
BioSpace
Amicus Therapeutics, announced the election and appointment of Eiry W. Roberts, M.D. to its Board of Directors.
BioSpace
May 6, 2021
BioSpace
Most Recently Served as Global Head of Rare Diseases at Sanofi Genzyme Brings Deep Expertise in Corporate Strategy, Business Development and Commercialization
Nick Paul
February 12, 2021
FierceBiotech
A phase 3 clinical trial of Amicus Therapeutics' late-onset Pompe disease prospect has missed its primary endpoint. Yet, Amicus looked past the failure of AT-GAA to improve statistically on Sanofi's market incumbent Lumizyme, zeroing in on details of the data to justify forging ahead with plans to seek approval of the candidate.
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