RARE Daily

FDA Grants Race’s Bisantrene Rare Pediatric Disease Designation

June 18, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Race Oncology Bisantrene Rare Pediatric Disease designation for the treatment of the rare blood cancer childhood acute myeloid leukemia.

The FDA grants Rare Pediatric Disease designation to incentive development of new treatments for serious or life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the United States. The RPDD program allows for a sponsor who receives an approval to qualify for a priority review voucher (PRV) that can be deemed to receive an expedited six-month priority review for any subsequent marketing application.

PRVs are potentially lucrative because they can be sold to another pharmaceutical company for use with another drug and indication. Since 2016, PRVs have been selling in the range of $110 million to $130 million each.

The RPD designation was granted in response to a submission from Race, which argued that childhood AML can be considered a substantively different disease to adult AML based on genetic markers disproportionately found in childhood AML and therefore that it constituted a rare pediatric disease.

“This is a game-changing outcome for Race that adds substantial value to the company,” said Race CEO, Peter Molloy. “To date, we have focused our clinical development plan on adult AML, which is the largest population of AML patients. Now, in parallel to the adult program, we plan to expedite a pediatric program directed towards securing the PRV.”

Photo: Peter Molloy, CEO of Race Oncology

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