Scioderm To Submit Rolling NDA To U.S. FDA For Zorblisa

DURHAM, N.C.--(BUSINESS WIRE)--Scioderm, Inc., a clinical-stage biopharmaceutical company focused on developing innovative therapies for treating diseases with high unmet need, including rare diseases, today announced that it has agreed with the U.S. Food and Drug Administration (FDA) to submit a rolling New Drug Application (NDA) submission for Zorblisa™ (SD-101), a proprietary and novel topical therapy for the treatment of blisters and lesions in patients with Epidermolysis Bullosa (EB), a rare genetic connective tissue disorder. A rolling submission is intended to accelerate the review process by enabling review of portions of the NDA by the FDA prior to submission of the complete application.

“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. We are thrilled at the prospect of making this novel therapy a reality for the patients and families living with the devastating effects of EB.”

Based on discussion with and agreement by the Agency, Scioderm plans to submit the non-clinical components of the NDA in the third quarter of 2015, followed by submission of the chemistry, manufacturing and control (CMC) components by early fourth quarter of this year. The FDA designated Zorblisa as a Breakthrough Therapy in 2013.

Scioderm is currently conducting a Phase 3 study in the U.S. and Europe, which is expected to support registration globally. The Phase 3 clinical program was initiated at U.S. and European sites in March 2015. The company plans to seek FDA and EMA approval of Zorblisa based on the development program agreed upon by both regulatory agencies and Scioderm.

Scioderm also announced that the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) 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. "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. We are thrilled at the prospect of making this novel therapy a reality for the patients and families living with the devastating effects of EB."

About Zorblisa^TM

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. Scioderm received Breakthrough Therapy designation for Zorblisa from the FDA, as well as orphan drug designation from the FDA and EMA.

About Epidermolysis Bullosa (EB)

Epidermolysis bullosa (EB) is a rare genetic disease leading to fragile skin susceptible to blistering and tearing. EB 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. There is currently no effective or FDA approved treatment for EB. Current standard of care consists solely of bandaging and bathing the open wounds to prevent infection and trying to manage patients’ pain.

About Scioderm, Inc.

Scioderm is a privately held, clinical-stage biopharmaceutical company focused on developing innovative therapies to address diseases with high unmet need, including rare diseases. The company’s lead therapy, Zorblisa™ (SD-101), is in Phase 3 development for treatment of the skin effects associated with Epidermolysis Bullosa (EB), a rare genetic connective tissue disorder. Scioderm was selected as a 2013 “Fierce Top 15” company by FierceBiotech, and considered one of the top 15 emerging companies in the biotech industry. The company is headquartered in Durham, North Carolina. Additional information about Scioderm can be found at www.sderm.com.

Forward Looking Statement

Except for the historical information contained herein, the matters discussed in this press release are forward-looking statements that involve risks and uncertainties, including: our dependence on third parties for the development, regulatory approval and successful commercialization of our products, the inherent risk of failure in developing product candidates based on new technologies, risks associated with the costs of clinical development efforts, as well as other risks. Actual results may differ materially from those projected. These forward-looking statements represent our judgment as of the date of the release. Scioderm disclaims any intent or obligation to update these forward-looking statements.