Updated on 27 January 2015
Dr Robert Ryan, CEO, Scioderm
Singapore: There is a clear trend throughout the pharmaceutical industry to focus efforts on unmet medical needs. Historically, orphan diseases have not received much drug development attention. That has changed recently especially in light of the incentives being promoted among the regulatory authorities in the US, Europe and few Asian countries. This trend is likely to continue with the regulators providing both incentives, including Breakthrough Therapy designation and Priority Review Vouchers, along with more collegial interactions in support of programs addressing unmet medical needs and pediatric diseases. The incentives offered in the "Breakthrough Therapy" program within the Food and Drug Administration (FDA) and the incremental exclusivity the European Medicines Agency (EMA) provided for pediatric drug development will continue to influence decisions that affect opportunity selection and risk/reward-based funding in the industry.
2014 was a very successful year for Scioderm with the company accomplishing multiple notable milestones. The company's Zorblisa was granted orphan designation in Europe for the treatment of EB, a rare genetic disease in which patients experience painful blisters and wounds over a substantial percentage of their bodies. Scioderm was granted multiple patents in the US.
As 2014 comes to a close, Scioderm is preparing for the initiation of its Phase 3 registration program, which will start at US and European investigational sites during the first quarter of 2015. This will support registration in both the US and EU. In addition, Scioderm expects to complete the Phase 3 study of Zorblisa by the end of 2015. Scioderm also looks to initiate a rolling NDA submission in the US and meet with the European Medicines Agency (EMA) to define the MAA submission plan for European registration.
Science and drug development opportunities are progressing at ever increasing speed. There is no reason to expect that the creativity of the industry is even nearing its peak. Consequently, even as we and others satisfy some unmet medical needs, other opportunities in the same or different therapeutic arenas will emerge.
The spirit of discovery and innovation has never been stronger and we are proud to be part of an industry dedicated to improving the health and lives of others. Just as we are anticipating progress in our program, the industry will continue pursuit of the programs in which significant investments of time, money and intellect show promise.