FDA grants rare pediatric disease, orphan drug designations to retinoblastoma treatment

The U.S. Food and Drug Administration has granted rare pediatric disease and orphan drug designations to for OXU-003 (Oxular Limited) for the treatment of retinoblastoma.

In preclinical models, the anti-tumor drug has been shown to be effective as a stand-alone therapy, as well as complementary to other current treatments.

“I am very pleased to see the FDA’s acknowledgement of the critical and urgent need to develop an effective, safer and easily accessible treatment for retinoblastoma which is such a devastating disease,” said Manoj Parulekar, Paediatric Consultant Ophthalmologist, Birmingham Children’s Hospital, said in a company news release. “Given the potential severe side effects associated with current treatments, which can have life-long impact on children’s lives, it is important that these patients have another treatment available to them.”

Read the full press release here.