SYDNEY, Sept. 9, 2020 /PRNewswire/ -- Clarity Pharmaceuticals, a clinical-stage radiopharmaceutical company focused on the treatment of serious disease, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Rare Paediatric Disease Designation (RPDD) to 64Cu-SARTATE™, a diagnostic for the clinical management of neuroblastoma.
Dr Alan Taylor, Clarity's Executive Chairman, commented, "We are very excited to have received the RPDD status for the diagnostic application of SARTATE™ in children with neuroblastoma. This comes shortly after Clarity has been granted RPDD for the treatment of neuroblastoma with 67Cu-SARTATE™ for the therapeutic application, announced on the 3rd of June 2020."
The FDA defines a "rare paediatric disease" (RPD) as a serious or life-threatening disease primarily affecting individuals aged 18 years or younger that impacts fewer than 200,000 people in the United States. The program is intended to facilitate development of new drugs and biologics for the prevention and treatment of RPDs.
Neuroblastoma most often occurs in children younger than 5 years of age and presents when the tumour grows and causes symptoms. It is the most common type of cancer to be diagnosed in the first year of life and accounts for around 15% of paediatric cancer mortality.[1] High-risk neuroblastoma accounts for approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rates at 40%-50%.[2]
Upon FDA marketing approval of 64Cu-SARTATE™ for neuroblastoma with RPD designation, Clarity would be eligible to receive a tradable Priority Review Voucher (PRV). The PRV shortens the FDA review period of a New Drug Application (NDA) for another product to an expedited period of six months, which is a huge benefit for drug developers. The voucher, if awarded, may be sold or transferred to another company. To date, PRVs have been sold for between US$67.5 million and US$350 million, with the most recent PRV being purchased by Merck from Lumos Pharma for a value of US$100 million in July 2020.[3]
"With 67Cu-SARTATE™, a therapeutic for the clinical management of neuroblastoma, also having been awarded RPDD status[4], Clarity may be eligible for two Priority Review Vouchers if both treatments get FDA approval," commented Dr Taylor.
"We have seen incredibly strong support from our collaborators and advisors in the development of SARTATE™ for neuroblastoma and are looking forward to the results from our US-based trial at the Memorial Sloan Kettering Cancer Centre[5]. It is evident that there is a large unmet need in the management and treatment of this devastating disease and we are aiming to improve outcomes for this important patient population with both the diagnostic and therapeutic applications of SARTATE™" said Dr Taylor.
References
About Clarity
Clarity is a personalised medicine company focused on the treatment of serious diseases. The Company is a leader in innovative radiopharmaceuticals, developing targeted therapies for the treatment of cancer and other serious diseases in adults and children.
www.claritypharmaceuticals.com
![Figure 1. Images show considerable reduction in lesion uptake (64Cu-SAR-bisPSMA PET) following 4 doses of 67Cu-SAR-bisPSMA (4GBq each; PET conducted in July 2024, approximately 14 months post-fourth cycle). Reduction in uptake (maximum standardised uptake value [SUVmax]) and tumour volume: 72.5% and 41.6%, respectively. New bone lesions identified (red circles) in the most recent image prior to the fifth dose. The fifth dose was delivered on 30 July 2024, with PSA having fallen 47.5% as of 24 September 2024 and trending downward. Post-treatment scans are pending.](https://mma.prnasia.com/media2/2531886/Images_show_considerable_reduction_lesion_uptake__64Cu_SAR_bisPSMA_PET__4_doses.jpg?p=medium600)
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