Epidermolysis Bullosa (EB) | Amicus Therapeutics

We are developing the topical therapy SD-101 for the treatment of skin blistering and lesions associated with Epidermolysis Bullosa (EB).

Epidermolysis Bullosa (EB)

We are developing the topical therapy SD-101 for the treatment of skin blistering and lesions associated with Epidermolysis Bullosa (EB).

  • EB 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.

Development Status

For more information about the Phase 3 study, read about our clinical trials.

A single-center, open-label Phase 2a study of SD-101 study was completed in in 8 EB patients with three major EB types, aged six months to nine years. In this single-arm study, SD-101 cream containing a 3% concentration of allantoin was applied to the entire body once daily for three months.

Following the completion of the Phase 2a study and subsequent interactions with the FDA, a multicenter Phase 2b study (SD-003) was conducted to further investigate SD-101 in 48 patients with three major EB types. Patients were randomized into one of three treatment arms: a higher concentration of active ingredient (SD-101 6%), the same concentration used in the Phase 2a study (SD-101 3%), or placebo.

All patients that completed the SD-003 study were eligible to continue to receive active therapy in the Phase 2 open-label extension study SD-004 which is currently underway.

In 2013, SD-101 became one of the first drug candidates to receive Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with EB. SD-101 also has orphan drug designation from the FDA and European Medicines Agency (EMA).

Bullosa (EB)

SD-101: Phase 3 Study SD-005

Study of Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa

Active, not recruiting.

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of SD-101 Cream in patients 1 month and older with a diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB who have a wound that meets specific study criteria as assessed by a healthcare professional. SD-101 (6%) or placebo will be applied topically, once a day to the entire body for a period of 90 days. Patients who complete the study will be eligible to enroll in an open-label extension Study (SD-006).

More information: www.clinicaltrials.gov: NCT01458119

SD-101: Open-Label Extension Study SD-004

Open Label Extension Study to Evaluate the Safety of SD-101 Cream in Subjects with Epidermolysis Bullosa

Ongoing, recruitment closed. Patients who completed the treatment period in the Phase 2b study (SD-003) of SD-101 were eligible to roll over into SD-004.

Open-label extension study to assess the continued safety of topically applied SD-101 Cream (6%) in subjects with Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB. SD-101 (6%) is applied topically, once a day to the entire body.

More information: www.clinicaltrials.gov: NCT01458119