FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Oxular

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Oxular has been granted rare pediatric disease and orphan drug designations by the US FDA for its proprietary drug, OXU-003.

According to an Oct. 12, 2020 press release, Oxular, a retinal therapeutics development company, has been granted rare pediatric disease and orphan drug designations by the US Food and Drug Administration (FDA) for its proprietary drug, OXU-003.

OXU-003 is currently under development for the treatment of retinoblastoma, a form of eye cancer that usually develops in early childhood. Current treatment options for retinoblastoma are invasive and require specialized facilities. OXU-003 consists of a proprietary anti-tumor drug that uses Oxular’s formulation and ocular administration technology to deliver a precise amount of drug adjacent to the primary ocular tumor.

“I am very pleased to see the US 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, pediatric consultant ophthalmologist, Birmingham (UK) Children’s Hospital, in the press 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.”


“We are delighted to receive these rare pediatric disease and orphan drug designations, which provide important momentum for our OXU-003 development program, as we expect to enter human clinical trials and generate data within the next two years,” added Thomas Cavanagh, CEO of Oxular, in the press release. “OXU-003 is a potential breakthrough therapy that utilizes Oxular’s core technology to maximize the opportunity for successful treatment while preserving quality of life for these young patients.”

Source: Oxular