Clarity Pharmaceuticals Announced the US FDA Grants Rare Pediatric Disease Designation to 64Cu-SARTATE™

September 9, 2020

Clarity Pharmaceuticals is a clinical-stage radiopharmaceutical company focused on the treatment of serious disease. Today they announced 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.

Clarity Pharmaceuticals Executive Chairman Dr. Alan Taylor 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 3^rd of June 2020.”

The FDA defines a “rare pediatric 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 the 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 tumor 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 pediatric 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

1. Nadja C. Colon and Dai H. Chung 2011, “Neuroblastoma”, Advances in Paediatrics, <https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3668791/>
2. Valeria Smith and Jennifer Foster 2018, High Risk Neuroblastoma Treatment Review, <https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6162495/>
3. Lumos Pharma Announces Sale of Priority Review Voucher 27 July 2020, <https://www.globenewswire.com/news-release/2020/07/27/2068182/0/en/Lumos-Pharma-Announces-Sale-of-Priority-Review-Voucher.html>
4. Clarity Pharmaceuticals Announces that the US FDA Grants Rare Paediatric Disease Designation to 67Cu-SARTATE™ for the Treatment of Neuroblastoma 3 June 2020, <https://www.claritypharmaceuticals.com/news/rpdd/>
5. ClinicalTrials.gov Identifier: NCT04023331 <https://clinicaltrials.gov/ct2/show/NCT04023331>