Rare Disease Report

EB Drug SD-101 Gets Rare Pediatric Disease Designation

MAY 31, 2017
Mathew Shanley
Per a press release, Rare Pediatric Disease designation was granted by the U.S. Food and Drug Administration to Amicus Therapeutics and for their drug SD-101, intended for the treatment of skin blistering and lesions associated with epidermolysis bullosa (EB).
EB is a group of congenital connective tissue diseases that manifests as blisters in the skin, or, occasionally the epithelial lining of other organs. It is often caused by a defect in the connectivity of the epidermis and dermis, and results in both friction and fragility of the skin. SD-101 was granted orphan drug designation in 2014, and was one of the first treatments to receive the FDA’s Breakthrough Therapy designation (2013). It is a topical cream that has shown the potential to improve the severe skin effects seen in EB patients.
To be granted Rare Pediatric Disease designation, a drug must be designed for the treatment of diseases that mostly affect children and adolescents ages 18 years or younger, and less than 200,000 patients in the U.S. The Rare Pediatric Disease designation for SD-101 covers the broad treatment of the rare disease.
If Amicus’ EB drug gets approved, the company will receive a Rare Pediatric Disease Priority Review Voucher which they can use on ANY future drug or sell it to another company. In the past, these vouchers have sold for as high as $350 million. Prices have since dropped considerably, as Sarepta sold theirs for $125 million.
“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,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc.
“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.”
The Phase 3 ESSENCE study of SD-101 is double-blind and placebo-controlled. More than 160 patients were enrolled, and each of them has documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.
Reporting of top-line data from the study is anticipated for the third quarter of 2017.

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