Retinoblastoma generally affects children before the age of three. It is rarely found in adults.
Cellceutix said the eye cancer is sight threatening and potentially fatal if not diagnosed early.
The designation provides Cellceutix an opportunity to apply for participation in the FDA’s rare pediatric disease priority review voucher program.
The program aims to incentivize development of drugs for neglected diseases where no sufficient therapies exist.
Under the program, a sponsor who secures an approval for a drug or biologic for a rare pediatric disease could qualify for a voucher which can be redeemed to receive a priority review of a marketing application for a different product. The recipient may sell or transfer the voucher.
Cellceutix CEO Leo Ehrlich said: "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 (removal) of the eye is often the only solution."
Kevetrin is a small molecule that induces the activation of the tumor suppressor protein p53.
p53 is encoded by the TP53 gene in humans and has been shown to play important roles in the homeostatic health of the human body by activating proteins needed to repair DNA.
It plays an important role in the life cycle of cells by inducing cell cycle arrest and apoptosis for maintaining cellular and genetic stability.