Retinoblastoma drug granted rare pediatric disease designation

Cellceutix Corporation announced that the U.S. FDA granted rare pediatric disease designation to kevetrin for the treatment of retinoblastoma.

Kevetrin is a small molecule compound that activates p53, often referred to as the “guardian angel gene” due to its crucial role in controlling cell mutations. It is currently in a Phase 1 clinical trial for patients with advanced solid tumors.

The designation permits application to the FDA's rare pediatric disease priority review voucher program, which can be redeemed to receive a priority review of a subsequent marketing application for a different product. The voucher may also be sold by the recipient to a third party. The program is intended to incentivize development of drugs for neglected diseases that lack adequate therapies. This designation supplements the orphan drug designation previously granted by the FDA to kevetrin for retinoblastoma and ovarian cancer.

"Receiving the rare pediatric disease designation strengthens our portfolio and adds to our belief that kevetrin has the potential to provide a meaningful therapeutic benefit to children and families affected by retinoblastoma, where enucleation of the eye is often the only solution," said Leo Ehrlich, CEO of Cellceutix. "We are putting plans in place to move ahead with this exciting project. We view the voucher program as very valuable in meeting areas of unmet medical need and certainly keep in mind the monetary component considering United Therapeutics recently agreed to sell its rare pediatric disease priority review voucher to a subsidiary of AbbVie for $350 million."