Scioderm Gets Accelerated Review of New Epidermolysis Bullosa Drug
July 16, 2015
Durham-based Scioderm Inc.,a biopharmaceutical company seeking innovative therapies for diseases with high unmet needs, will get an accelerated review of its drug that treats lesions and blisters in a rare genetic disorder called Epidermolysis Bullosa (EB).
The U.S. Food & Drug Administration and the company have agreed that it should submit a rolling New Drug Application (NDA) for the treatment, Zorblisa, a topical therapy that completed a Phase 2 study in EB patients. Data showed faster wound healing and closure of chronic wounds.
EB leads to fragile skin susceptible to blistering and tearing. It is chronic, debilitating and potentially disfiguring and patients with EB have painful blisters affecting a substantial percentage of their bodies that can lead to infection and scarring.
There are approximately 20,000-30,000 people living with EB in the United States, and 300,000-400,000 worldwide. Currently, there is no effective FDA approved treatment for the disease, which is treated by bandaging and bathing the open wounds and trying to manage patient pain.
Zorblisa is a proprietary topical therapy being developed for the treatment of patients with EB. Scioderm recently completed a successful Phase 2 study (SD-003) in EB patients, which was a double-blind, randomized, placebo-controlled, dose-response trial evaluating the efficacy and safety of different dosage strengths of Zorblisa. The data showed acceleration in wound healing and closure of chronic wounds.
The company says it expects data from a Phase 3 trial as soon as early 2016 and plans to seek approval and commercialize soon thereafter.
The FDA designated Zorblisa as a Breakthrough Therapy in 2013. It also has orphan drug designation from both the FDA and EMA. Both designations are intended to speed consideration of much needed treatments for rare diseases.
Scioderm also said that the Pediatric Committee of the European Medicines Agency has issued a positive opinion on the company’s Pediatric Investigation Plan (PIP) for Zorblisa. A PIP is part of the EMA approval process and must be accepted prior to a submission of a Marketing Authorization Application (MAA) in the European Union.
“The initiation of our NDA submission for Zorblisa marks a significant milestone for the EB community, and we look forward to completing the submission and working with the Agency on our mutual goal of providing treatment options to EB patients as quickly as possible,” said Robert Ryan, Ph.D., president and chief executive officer, Scioderm in a press release.
“Further, the acceptance of our PIP by the EMA is aligned with our U.S. plan and enables a single global development program for Zorblisa.”
WRAL TechWire Insiders can read the story of Bret Kopelan’s daughter, Rafaella, who suffers from the disease. Kopelan hopes this treatment will help her. Read more on WRAL TechWire Insiders here.
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