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Scioderm Receives FDA Breakthrough Therapy Designation For Epidermolysis Bullosa

April 30, 2013

Scioderm announces on April 29, 2013, that the company’s investigational topical cream, SD-101, receives FDA’s Breakthrough Therapy Designation for the treatment of patients with inherited Epidermolysis Bullosa (EB). Scioderm is a pharmaceutical company focusing on the development of drugs for the treatment of chronic diseases of the skin. The company is headquartered in Durham, North Carolina.

EB is a rare genetic connective tissue condition that involves very fragile skin that blisters or tears with the slightest trauma or friction. EB patients are known as “Butterfly children” due to the fragile nature of the skin, similar to the fragility of the wings of a butterfly. There is currently no cure or effective treatment, outside of daily wound care, pain management, and preventative bandaging.

The granting of this designation is based on clinical data from a previously conducted open-label Phase II study with topical administration of SD-101 in children with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB. The primary outcome measurements is the assessment of target wound reduction and closure, and the reduction in Body Surface Area (BSA) coverage of lesions and erosions. In the clinical trial after daily treatment of SD-101, there is :

•   Complete closure of 88% of target chronic lesions within one month
•   57% reduction in BSA coverage after 3 months.

10 Breakthrough Therapy Designations

The 10 Breakthrough Therapy Designations granted for drugs that have been identified by the sponsor companies (as of 04/30/13) are as follows :

• Two are awarded to Vertex Pharmaceuticals for expanding the uses of its Cystic Fibrosis orphan drug Kalydeco

• Three are awarded to Pharmacyclics and Janssen Research & Development for the cancer therapy drug Ibrutinib for relapsed or refractory Mantle Cell Lymphoma (MCL – orphan designation), Waldenstrom’s Macroglobulinemia (WM), and Chronic Lymphocytic Leukemia (CLL – orphan designation) or Small Lymphocytic Lymphoma (SLL) with deletion of the short arm of chromosome 17

• One is awarded to Novartis for cancer non-orphan drug LDK378 for ALK+ Non-Small Cell Lung Cancer

• One is awarded to Pfizer for breast cancer non-orphan drug Palbociclib

• One is awarded to Merck for cancer non-orphan drug Lambrolizumab for Melanoma

• One is awarded to Bristol-Myers Squibb for experimental, non-orphan three-drug regimen for Hepatitis C

• One is awarded to Scioderm for investigational product SD-101 for Epidermolysis Bullosa (EB).


Dystrophic Epidemolysis Bullosa Research Association of America (debRA)

Scioderm Pipeline

Scioderm’s “What is Epidermolysis Bullosa (EB) ?”.

Please Note #1: The Orphan Druganaut Blog has decided to include a Post on Scioderm’s investigational product SD-101  even though it isn’t an orphan drug since the Blog has been following the new FDA Breakthrough Therapy designation from the beginning of 2013.

Please Note #2: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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