News Releases

Thursday, August 31, 2017

Isakson Applauds Approval of First Gene Therapy Treatment in U.S.

Speedy treatment approval will help child cancer patients thanks to extension of FDA program secured by Isakson

WASHINGTON – U.S. Senator Johnny Isakson, R-Ga., today applauded the Food and Drug Administration’s (FDA) approval of the first cell-based gene therapy available in the United States. The approval came thanks to Isakson’s work on the Advancing Hope Act, which extended the Rare Pediatric Disease Priority Review Voucher Program and was included in the 21st Century Cures Act, signed into law in December 2016.

This new therapy—called Kymriah—was approved for certain pediatric and young adult patients suffering from a form of acute lymphoblastic leukemia – the most common childhood cancer in the United States – and is an innovative, new therapy that reprograms a patient’s own cells to attack a deadly cancer.

FDA officials called Isakson Wednesday to relay the news of the agency’s approval of the groundbreaking therapy.

“This type of therapy is exactly what we had in mind when I began working for the Advancing Hope Act, which was ultimately approved and extended in last year’s 21st Century Cures legislation,” said Isakson. “When I heard this wonderful news directly from the FDA yesterday, I thanked them and told them to get it on the market, because it’s time to start saving kids’ lives.”

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Isakson’s commitment to helping find effective treatments for rare pediatric diseases began in 2004, following a visit from Alexa Rohrbach, a young Georgian suffering from a rare cancer who came to see Isakson to ask for his help in finding a cure. Alexa passed away in 2008, but Isakson said that her memory is “a reminder of why getting a result on bills like this one is as critical as anything we could ever do in the United States Senate.” He shared Alexa’s story on the Senate floor in 2016 to urge his colleagues to support an extension of the priority review program.

The newly approved therapy will be used to treat acute lymphoblastic leukemia, a cancer of the bone marrow and blood that progresses quickly and is the most common childhood cancer in the United States. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with acute lymphoblastic leukemia each year. Kymriah is approved for use in pediatric and young adult patients with B-cell acute lymphoblastic leukemia and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15 to 20 percent of patients.

The clinical review was coordinated by the FDA’s Oncology Center of Excellence, which was established under the 21st Century Cures Act, while the Center for Biologics Evaluation and Research conducted all other aspects of review and made the final product approval determination. Kymriah received a Rare Pediatric Disease Priority Review Voucher.