Amicus Therapeutics, Inc.
0HF9.L
London Stock Exchange17.09
USD
+2.63
- -
Amicus Therapeutics, Inc.
0HF9.L
(London Stock Exchange)
Recent
price
17.09
P/E
ratio
- -
div
yld
- -
ROIC.AI
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in mil. unless spec.- Similar Companies
- Business
- Amicus Therapeutics, Inc. is a biopharmaceutical firm dedicated to the identification, advancement, and provision of therapeutic interventions for uncommon disorders. Its commercial portfolio includes Galafold, an oral precision medication prescribed for adults with a confirmed diagnosis of Fabry disease, specifically those with an amenable galactosidase alpha gene variant, as evidenced by in vitro assay data. Furthermore, the company has successfully introduced Pombiliti and Opfolda, a two-component treatment regimen for adults living with late-onset Pompe disease who are not demonstrating improvement with their existing enzyme replacement therapy. The company’s development pipeline extends to pioneering gene therapies for several severe neurological conditions. This includes AT-GTX-502 for CLN3 Batten disease, currently undergoing a Phase 1/2 clinical study to assess the safety and effectiveness of a single intrathecal administration. Amicus is also advancing a gene therapy program for CDKL5 deficiency disorder, a condition involving a gene on the X-chromosome responsible for regulating essential proteins in normal brain development. Collaborations underpin its research and development efforts, notably with Nationwide Children's Hospital and the University of Pennsylvania. Amicus Therapeutics, Inc. was established in 2002 and maintains its headquarters in Philadelphia, Pennsylvania.