A regulatory agency in Europe has granted “orphan designation” to a product discovered at Cincinnati Children’s that could prevent a serious lung complication that often develops in extremely premature infants.
The product - recombinant human surfactant protein D – is being developed by Airway Therapeutics, a start-up company co-funded by Cincinnati Children’s and CincyTech, a public-private seed-stage investor. The product is based on research led by Jeffrey Whitsett, MD, co-director of the Perinatal Institute at Cincinnati Children’s.
Surfactant D would be an improvement to existing synthetic forms of surfactant that have saved the lives of many thousands of premature infants born with under-developed lungs. Early data shows that the new product appears effective at preventing bronchopulmonary dysplasia (BPD), an inflammation and scarring of lung tissues that often occurs when infants spend long periods using a ventilator.
On April 11, a committee of the European Medicines Agency granted orphan designation to the surfactant D product. The designation supports further development of products made to treat life-threatening rare diseases that lack other satisfactory treatment options.
Achieving this status is a significant step in the long drug approval process. It helps pave the way toward clinical trials in Europe and gives the company 12 years of marketing exclusivity in the European Union if the treatment makes it to market.
The company hopes to begin clinical testing in Europe in 2016. It also plans to begin seeking regulatory approval for US clinical trials.
Read a Cincinnati Enquirer article with more details about the product and the start-up company.