Sen. Tim Scott leads bicameral letter urging FDA to reconsider gene therapy decision

WASHINGTON— Today, U.S. Senator Tim Scott (R-SC) penned aletterto the U.S. Food and Drug Administration (FDA) urging the agency to reconsider and reverse a prior decision denying a Pediatric Rare Disease Priority Review Voucher (PRV) for a gene therapy treating sickle cell disease (SCD). Sen. Scott was joined on the letter by Representatives Gus Bilirakis (R-FL-12), Mike Kelly (R-PA-16), Rick Allen (R-GA-12), and Buddy Carter (R-GA-1).
In the letter, lawmakers highlight the devastating toll of sickle cell disease, which affects approximately 100,000 Americans, more than 90% of whom are African American, and underscore the importance of advancing curative treatments.
“We write regarding the administration of the Food and Drug Administration’s (FDA’s) Pediatric Rare Disease Priority Review Voucher (PRV) program, particularly the decision by the previous administration to deny a PRV for a one-time administered novel curative gene therapy that treats sickle cell disease,”the lawmakers wrote.
They continued,“This chronic, debilitating disease causes severe pain episodes often resulting in hospitalization, progressive organ damage, and serious acute events like strokes… The approvals in December 2023 of two curative gene therapies represented a transformative moment for Americans…living with SCD.”
The lawmakers also argue the FDA’s denial was based on a flawed interpretation of statute, writing that the agency“applied a newly created, extra-statutory definition of “active ingredient’”to determine that the therapy was not eligible for a PRV.
“The previous administration ignored this guidance, inexplicably choosing to treat the modified cell types of patients with Betathalassemia and those with SCD as the same ‘active ingredient,’”they wrote, noting that this approach conflicts with both FDA precedent and congressional intent behind the program.
They warned that maintaining this interpretation could have far-reaching consequences for innovation.“Upholding this arbitrary decision… risks jeopardizing the viability of an industry intent on creating novel treatments for pediatric patients with sickle cell disease and other rare, severe genetic diseases,”the lawmakers continued.
The letter emphasizes that the PRV program, established by Congress in 2012, was designed to incentivize development of treatments for rare pediatric diseases without taxpayer cost, and that reversing the decision would restore those incentives.“Revisiting, and ultimately reversing, this decision… will further support [ongoing efforts to advance gene therapies]… and do so at no cost to the federal government,”they wrote.
The lawmakers conclude by urging FDA leadership to take a fresh look at the decision.“We are grateful for your leadership… and we kindly ask that you take this opportunity to correct this decision made by the previous Administration,”they concluded.
Read the full letterhere.
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