News and Press Releases.
New Pediatric Cancer Drug Approved by FDA
Creating Hope Act Sponsors React to FDA Approval of Pediatric Cancer Treatment
2012 Law Established Voucher Program that Enabled Development of Treatment
WASHINGTON, DC – Today the Food and Drug Administration announced it has approved the first pediatric cancer drug due to the voucher program that became law under the Creating Hope Act. The drug, Unituxin, developed by United Therapeutics, designed to treat neuroblastoma, is only the third third drug to receive initial FDA approval for a pediatric cancer in the last 20 years. The Creating Hope Act was signed into law in 2011, and was sponsored by Representative Michael McCaul, Representative G.K. Butterfield, and Representative Chris Van Hollen.
McCaul: "The scientists and doctors who developed this drug deserve credit and recognition for this amazing achievement. Too many children suffer from neuroblastoma and for them and their families, the development of the first FDA approved treatment in decades is welcome news. I am proud that the Creating Hope Act played an important role in this process. The voucher program established by this law has once again demonstrated its value. I look forward to working with my colleagues to make this program permanent for children with all rare diseases, such as pediatric cancer and sickle cell disease.
Butterfield: "I'm pleased to learn that the Creating Hope Act is fulfilling its purpose of advancing drugs for rare pediatric diseases to market," said Congressman Butterfield. "Since its enactment, less than four years ago, we've already seen two pediatric drugs receive approval by the FDA. I look forward to more drugs gaining approval and more children receiving the treatment they need.”
Van Hollen: ""The Creating Hope Act was designed to spur both lifesaving cures for children and new innovations that help American companies grow and create jobs," said Congressman Van Hollen. "I'm glad a Maryland business is the latest to benefit from this program to kick-start investment in treatments and cures for children and families affected by cancer."
The Creating Hope Act provides market incentives to pharmaceutical companies to develop new drugs for children with rare pediatric diseases, such as childhood cancers and sickle cell. This is the second voucher awarded under the law.
The Creating Hope Act:
- Establishes the Priority Review Voucher program to include treatments for pediatric rare diseases, such as childhood cancers.
- Offers unlimited transferability of vouchers to create a more easily traded asset.
- Provides greater certainty to sponsors that the drug, if approved, will qualify for a voucher, by permitting them to seek a designation from FDA before they submit their new drug application.