This designation is intended to encourage the development of therapies for rare and serious diseases, which are expected to outperform existing alternatives. Among its benefits are seven years of market exclusivity, exemption from FDA application fees, the agency’s assistance in clinical trial design, and tax credits for trial expenses.
“The granting of Orphan Drug Designation in the US highlights the significant need for a drug that could improve the treatment of EB, a debilitating disease that affects the life of half a million people in the US,” Paolo Galfetti, said in a press release. Galfetti is CEO of APR Applied Pharma Research, the treatment’s developer.
EB is characterized by skin blistering that may affect other parts of the body. Its symptoms include itching, pain and skin scars, as well as persistent skin inflammation and infections.
APR-TD011, specifically developed for EB, is administered as an easy-to-use spray formulation. This allows the treatment of skin wounds, while avoiding skin contact and contamination.
The therapy contains hypochlorous acid, a broad-spectrum and fast-acting antimicrobial agent. Its low pH and anti-oxidizing properties enable it to stop skin infections. APR-TD011 may decrease inflammation by blocking the NF-kB pro-inflammatory pathway and inactivating enzymes called matrix metalloproteases — key triggers of inflammation that regulate the migration of inflammatory cells.
In addition, multiple mechanisms of APR-TD011 may result in optimal conditions for the growth of new cells, supporting faster wound healing. Along with an intended reduction in the risk of scars and in itching and pain, these benefits may improve the quality of life of people with EB, according to the Switzerland-based company.
APR-TD011 was developed using the company’s nanotechnology platform, called Tehclo, which provides consistent quality in the production of hypochlorous acid solutions, APR said.
“We believe that APR-TD011 holds great promises for EB patients and their families and the Orphan status will help APR to find expedited pathways to fruition,” Galfetti said.
“This designation is also an important milestone and step forward in APR’s evolution as we advance our pipeline targeting rare diseases,” Galfetti added.