FDA Grants Rare Pediatric Disease Designation to Clarity’s Drug as a Diagnostic for Neuroblastoma

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to Clarity Pharmaceuticals’ 67Cu-SARTATE as a diagnostic for the clinical management of neuroblastoma.

The designation comes three months after the FDA granted Rare Pediatric Disease designation to 67Cu-SARTATE as a therapy for the clinical management of neuroblastoma.

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 percent of pediatric cancer mortality. High-risk neuroblastoma accounts for approximately 45 percent of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest five-year survival rates at 40 percent to 50 percent.

Clarity’s lead candidate, 67Cu-SARTATE, is a novel radiopharmaceutical compound for treating cancer. It employs personalized medicine to better diagnose and treat cancers. SARTATE has the ability to deliver isotopes of copper directly to cancers. It utilizes Copper-64 and PET imaging to diagnose where the drug goes in the body and confirm targeting. For therapy, it uses Copper-67, which kills cancer in a localized manner and has more favorable characteristics than alternative isotopes on the market, making it safer and more effective to use, the company said.

The FDA defines a rare pediatric disease 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 these conditions.

Upon FDA marketing approval of 67Cu-SARTATE for use as a diagnostic for the clinical management of neuroblastoma with Rare Pediatric Disease designation (RPDD), Clarity may be eligible to receive a Priority Review Voucher. The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

“With 67Cu-SARTATE, a therapeutic for the clinical management of neuroblastoma, also having been awarded RPDD status, Clarity may be eligible for two Priority Review Vouchers if both treatments get FDA approval,” said Alan Taylor, executive chairman of Clarity Pharmaceuticals. “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 Center. 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.”

Photo: Alan Taylor, executive chairman of Clarity Pharmaceuticals

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.