Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious illnesses, is pleased to announce that the Food and Drug Administration (FDA) in the United States has granted the designation of disease rare pediatric (or RPDD according to the English acronym) with 67 Cu-SARTATE ™, a therapy intended for the clinical treatment of neuroblastoma.

The FDA defines as "rare pediatric disease" (or RPD according to the English acronym) a serious or fatal disease mainly affecting people under the age of 18 and whose prevalence in the United States is less than 200,000 cases. The objective of the program is to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

Neuroblastoma most commonly occurs in children under the age of 5 and appears after the tumor grows and causes symptoms. It is the most frequently diagnosed type of cancer in the first year of life, and accounts for almost 15% of childhood cancer deaths. [1] High-risk neuroblastomas represent approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rate, 40% -50%. [2]

After the FDA authorizes the marketing of 67 Cu-SARTATE ™ for neuroblastoma with RPD designation, Clarity may obtain a right of access to a priority review voucher (PRV), which can be used so the FDA reviews a new drug request for another product in an accelerated six-month period. This access right can also be sold or transferred and, to date, access rights have been sold for between US $ 67.5 million and US $ 350 million.

Dr. Alan Taylor, Executive Chairman of Clarity, said, "The decision by the FDA to grant RPDD to 67 Cu-SARTATE ™ for the treatment of neuroblastoma, which follows the decision to grant it the drug designation an orphan, underlines the urgent need for better treatments for this terrible disease and testifies to the considerable work that we have accomplished to date within the framework of this therapy.

The strategies currently applied in the treatment of neuroblastoma are limited, especially in the advanced stage of the disease, and the prognosis of patients with high-risk neuroblastoma remains unfavorable. Our Clarity team and collaborators around the world are working to improve these results.

We are particularly pleased with the development of SARTATE ™ in the field of neuroblastoma and look forward to the results of our phase 1/2 trial based in the United States [3] . We hope that the granting of RPDD will bring us closer to our ultimate goal of developing better treatments for children and adults with cancer. "

References

1. Nadja C. Colon and Dai H. Chung 2011, "Neuroblastoma", Advances in Pediatrics , 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. ClinicalTrials.gov Identifier: NCT04023331
https://clinicaltrials.gov/ct2/show/NCT04023331

About Clarity

Clarity is a personalized medicine company focused on the treatment of serious illnesses. It is a leader in innovative radiopharmaceuticals and develops targeted therapies for the treatment of cancer and other serious diseases in adults and children.

 

Source: prnewswire.com

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