Amicus Therapeutics Receives Rare Pediatric Disease Designation for SD-101 for Patients with Epidermolysis Bullosa

June 6, 2017

 Amicus Therapeutics, Inc.(link is external) (Nasdaq:FOLD), has received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for the novel topical medicine SD-101 for the treatment of epidermolysis bullosa (EB). The Rare Pediatric Disease designation for SD-101 covers the broad treatment of EB. In addition to the Rare Pediatric Disease designation, SD-101 has Orphan Drug designation and was one of the first treatments to receive the FDA’s Breakthrough Therapy designation.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the U.S. If a new drug application (NDA) for SD-101 is approved, Amicus is eligible to receive a priority review voucher that may be sold or transferred.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “This Rare Pediatric Disease designation is significant in its broad coverage for the treatment of EB, and adds to our previous Orphan Drug and Breakthrough Therapy designations for SD-101 from the FDA. We believe that these important designations highlight the urgent need for a treatment for this devastating rare disease. SD-101, which we are developing for all three major types of EB, was the first drug to enter Phase 3 development for EB, and has the potential to be the first FDA-approved therapy. SD-101 has the potential to provide meaningful clinical benefit to patients and their caregivers and we eagerly await the results of our Phase 3 study.”

Amicus is on track to report top-line data from the Phase 3 ESSENCE study of SD-101 during the third quarter of 2017. ESSENCE is a double-blind, placebo-controlled registration study that completed enrollment of 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 patients completing the 3-month primary treatment period have elected to continue in the open-label extension study.

About Epidermolysis Bullosa (EB)
EB(link is external) is a rare, genetic disorder that manifests as blistering or erosion of the skin, and, in some cases, the epithelial lining of other organs. EB is chronic, potentially disfiguring, and in some cases fatal. Patients with EB have painful wounds and blisters that can lead to infection and scarring. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma. There is currently no approved treatment for EB. Current standard of care consists of pain management and the bandaging and cleaning of open wounds to prevent infection.

About Amicus Therapeutics 
Amicus Therapeutics(link is external) (Nasdaq:FOLD) is a 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(link is external) as a monotherapy for Fabry disease, SD-101(link is external) for epidermolysis bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

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