The family of a little girl who was born with a rare and severe skin disorder is excited to hear about the development of a drug that just might be the answer to this incurable condition.
When Rafaella (Rafi) Kopelan was born in November 2007, she had no skin on both her feet and at the back of one hand. Not long after, her skin started to blister and tear. Rafi, who was delivered two weeks late, was rushed to the neonatal intensive care unit.
After nine days of monitoring and checks, doctors tested Rafi for the rare and incurable genetic disorder Epidermolysis Bullosa (EB)
Since then, the Kopelans have dedicated much of their time to try to find a cure for their little girl's condition, but have not found any until latest news about an accelerated review of a drug that treats skin lesions caused by the rare disorder came about.
Scioderm Inc., a Durham-based biopharmaceutical company that aims to find innovations in therapies for the highly unmet needs of diseases, completed its Phase 3 study on EB patients, and has agreed with the US Food and Drug Administration (FDA) to pass a rolling New Drug Application (NDA) for its topical treatment Zorblisa.
The pharmaceutical company found positive results in the faster healing of wounds and closing of chronic wounds.
The drug could be most useful in EB patients where the skin becomes very fragile. EB patients experience painful blisters that may cause scarring and infection. EB is chronic, debilitating and may be disfiguring.
Zorblisa is the first drug to have reached Phase 3 in its development for the cure of EB. Scioderm agreed to have detailed discussions with the FDA throughout the development of the drug.
"The initiation of our NDA submission for Zorblisa marks a significant milestone for the EB community," said Dr. Robert Ryan, PhD, president and CEO of Scioderm. The CEO added how much they look forward to working with the FDA and with EB patients in their goal to provide treatment options in the quickest way possible.
Dr. Ryan is also working with the Kopelans and regularly speaks with Rafi's dad, Brett Kopelan, to spread awareness of the successful clinical trial.
Brett, who had to work from home for the first three years since Rafi was born, is the Executive Director of Dystrophic Epidermolysis Bullosa Research Association (DebRA).
Rafi's mom meanwhile had to stop working to be able to attend to Rafi's needs. Preparation time alone to get Rafi going for the day takes about 90 minutes - bathing, applying expensive cream and putting on protective bandages to keep her skin from blistering and getting infected.
While the Kopelans wait for Zorblisa to be approved, now eight-year-old Rafi goes to school in Manhattan, plays on swings, rides her bike and plays with the family's dog - just with that whole lot of extra time and tending to.
