On May 19, 2020 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for 64Cu-SARTATE as a diagnostic agent for the clinical management of neuroblastoma (Press release, Clarity Pharmaceuticals, MAY 19, 2020, View Source [SID1234558240]).

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"This milestone comes shortly after the FDA granted Clarity ODD for 67Cu-SARTATE, a therapy for the treatment of neuroblastoma," commented Dr Alan Taylor, Clarity’s Executive Chairman. "The grant of both diagnostic and therapeutic ODDs highlights the importance of work conducted by our Team in the field of theranostics and indicates the support for the development of SARTATE for children with cancer."

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 mortality1. 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

FDA grants ODDs to facilitate the development of investigational therapies intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the United States. The designation allows Clarity Pharmaceuticals to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; potential tax credits on US clinical costs; exemptions from certain administrative FDA fees; and eligibility for grants to fund future clinical work.

Dr Taylor said, "We are very excited about receiving ODDs for both the diagnostic and therapeutic applications of SARTATE in neuroblastoma and our Team is looking forward to progressing the development of both 64Cu-SARTATE and 67Cu-SARTATE in some of the leading cancer centres in the U.S. with the recruitment to our theranostic US-based Phase 1/2 trial3 expected to commence shortly."

References
Nadja C. Colon and Dai H. Chung 2011, "Neuroblastoma", Advances in Pediatrics, <View Source>
Valeria Smith and Jennifer Foster 2018, "High Risk Neuroblastoma Treatment Review", Children, <View Source>
ClinicalTrials.gov Identifier: NCT04023331