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Amicus Therapeutics Completes Enrollment in Essence Phase 3 EB Clinical Study

Amicus Therapeutics Completes Enrollment in Essence Phase 3 EB Clinical Study

Amicus Therapeutics, a global biotechnology company, has completed enrollment in the ongoing Phase 3 clinical study (ESSENCE) of the novel topical medicine SD-101 for people with all 3 major types of epidermolysis bullosa (EB) (Simplex, Recessive Dystrophic and Junctional non-Herlitz EB). With the achievement of full enrollment, top-line data from this study are expected in the third quarter of 2017.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, "The completion of enrollment in our global Phase 3 ESSENCE study of SD-101 for epidermolysis bullosa is a significant accomplishment for our team at Amicus as well as for the EB community. This is the most advanced clinical study for EB, and we look forward to announcing top-line data from this study in the third quarter of this year."

The FDA has granted Breakthrough Therapy designation for SD-101 based on results from a Phase 2a study for the treatment of lesions in people with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types.

John C. Browning, Chief of Dermatology at Children's Hospital of San Antonio, stated, "The full enrollment in this Phase 3 trial is a significant step forward as we look to advance new wound healing treatments for EB. With no currently approved treatment options, there is an urgent need among people living with EB, as well as their caregivers. There has been tremendous commitment among patients and families, advocacy organizations and study investigators in working alongside Amicus to raise awareness of and drive enrollment in this important study."

The ESSENCE Study is a Phase 3 double-blind, placebo-controlled study that enrolled more than 160 patients who have a documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB. To date, more than 95 percent of participants completing the 3-month primary treatment period have elected to continue in the open-label extension study.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus' lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare conditions.

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