Often called “butterfly children,” pediatric EB patients have extremely fragile skin—much like the wings of a butterfly. There is no cure or effective treatment for EB, but a novel topical is about to enter Phase III trials, and, if it passes muster, it will be fast-tracked by the US Food and Drug Administration (FDA).
Scioderm Inc will 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 EB. A rolling submission is intended to accelerate the review process by enabling review of parts of the NDA by the FDA prior to submission of the complete application.
Based on discussion with and agreement by the FDA, Scioderm plans to submit the nonclinical 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.
Zorblisa was the first drug to ever receive the FDA’s breakthrough designation, and is the first drug to ever enter Phase 3 development for the treatment of EB.
Zorblisa is a novel topical therapy that has demonstrated target wound reduction and closure, and reduction in body surface area coverage, of blisters and lesions over 3 months in children with EB. In a Phase 2 study, Zorblisa was applied over the entire body area daily for 3 months. It was well-tolerated and resulted in complete closure of 88% of target chronic lesions within 1 month, in addition to a 57% reduction in body surface area coverage of blisters and lesions.
Scioderm is currently conducting a Phase 3 study in the US and Europe. The Phase 3 clinical program was initiated at US and European sites in March 2015. The company plans to seek FDA and European Medicines Agency (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 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.
EB is a rare genetic connective tissue disorder. Its prevalence is between 20,000 and 30,000 in the US, and between 300,000 and 400,000 worldwide. The condition is extremely painful, and the blisters often cover a substantial part of children’s bodies and can lead to infection and scarring. Patients use Vaseline or Aquaphor and special bandages to protect their bodies. In its more severe form, EB can lead to deformities including fusion of the fingers and toes, secondary skin infections, sepsis, and even death.