CRANBURY, N.J., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced financial results for the third quarter ended September 30, 2020. The Company also summarized recent program updates and reiterated its full-year 2020 guidance.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, During the third quarter, we made tremendous progress advancing our mission for patients and are on track to achieve our 2020 key strategic priorities, including our global Fabry commercial launch, Pompe late-stage development program, and advancing our industry-leading gene therapy pipeline. Through these efforts, we remain strongly positioned to achieve our vision of delivering groundbreaking new medicines and hopefully, one day, cures for people living with rare diseases.

Corporate Highlights

Third Quarter 2020 Financial Results

1 Full reconciliation of GAAP results to the Companys non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.

2020 Financial Guidance

2 A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure.

Anticipated Milestones by Program

Galafold (migalastat) Oral Precision Medicine for Fabry Disease

AT-GAA for Pompe Disease

Gene Therapy Portfolio

About Galafold

Galafold (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable GLA variants. In these patients, Galafold works by stabilizing the bodys own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in over 40 countries around the world, including the U.S., EU, U.K., Japan and others.

U.S. INDICATIONS AND USAGEGalafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow on Twitter and LinkedIn.

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Amicus Therapeutics Announces Third Quarter 2020 Financial Results and Corporate Updates - Citybizlist