On October 12, 2020 Oxular Limited ("Oxular"), a leading retinal therapeutics development company, reported it has received both Rare Paediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (U.S. FDA) for OXU-003, the Company’s proprietary drug in development for the treatment of retinoblastoma (Press release, Oxular, OCT 12, 2020, View Source [SID1234568355]).

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Retinoblastoma is a rare form of eye cancer that usually develops in early childhood, typically before the age of five. This form of cancer develops in the retina, which is the specialised light-sensitive tissue at the back of the eye that detects light and colour. The most common first sign of retinoblastoma is a visible whiteness in the pupil called "cat’s eye reflex" or leukocoria. Other signs and symptoms of retinoblastoma include crossed eyes or eyes that do not point in the same direction (strabismus), a change in eye colour, redness, soreness, or swelling of the eyelids, and blindness or poor vision in the affected eye or eyes.

Retinoblastoma is often curable when it is diagnosed early. However, if it is not treated promptly, this cancer can spread beyond the eye to other parts of the body. This advanced form of retinoblastoma can be life-threatening.

Current treatment options for retinoblastoma include systemic chemotherapy and intra-arterial chemotherapy, which are very invasive procedures and require specialised facilities and hospital stay. Intra-arterial chemotherapy is only available in select centres. These treatments can lead to significant side effects for patients, including neurocognitive impairment, loss of vision and hearing, and life-threatening infections (sepsis) from low blood counts (neutropenia).

Oxular’s OXU-003 programme for the treatment of retinoblastoma consists of a proprietary anti-tumour drug which utilises Oxular’s formulation and ocular administration technology to safely deliver a precise amount of drug adjacent to the primary ocular tumour (local or targeted chemotherapy). OXU-003 has been shown to be effective as a stand-alone therapy in preclinical models and is complementary with other agents currently used to treat retinoblastoma. Oxular’s minimally invasive local therapeutic approach is intended to be less risky compared to current treatments, is expected to spare patients from related side effects while preserving vision and can be administered by an ophthalmic surgeons in standard operating theatres without the need of specialised equipment.

Mr. Manoj Parulekar, Paediatric Consultant Ophthalmologist, Birmingham Children’s Hospital, commented:

"I am very pleased to see the U.S. 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. 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."

Thomas Cavanagh, Chief Executive Officer of Oxular commented:

"We are delighted to receive these Rare Paediatric 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. OXU-003 is a potential breakthrough therapy that utilises Oxular’s core technology to maximise the opportunity for successful treatment while preserving quality of life for these young patients."

The U.S. FDA grants Rare Paediatric and Orphan Drug designations to drugs intended for the treatment of rare diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the U.S. If a future New Drug Application (NDA) for OXU-003 is approved, Oxular is eligible to receive a Priority Review Voucher that may be sold or transferred.

The first NDA applicant to receive FDA approval for a particular active moiety to treat a particular disease with FDA Orphan Drug designation is entitled to various incentives of the Orphan Drug Act (ODA), including tax credits for qualified clinical testing, waiver of NDA / biologics license application (BLA) user fees, and eligibility for a seven-year exclusive marketing period for that drug and use upon marketing approval.