The U.S. House of Representatives votes to reauthorize the Creating Hope Act as part of the 21st Century Cures, H.R. 6.!

We are thrilled to report that in June, 2015, The U.S. House of Representatives passed 21st Century Cures, H.R.  6, and with it, the reauthorization of the Creating Hope Act, otherwise known as the Rare Pediatric Disease Priority Review Voucher Program, 21 U.S.C. Sec 360ff.

A thank you from all sick children to Chairman Upton,  Congresswoman DeGette, and Congressmen Butterfield and McCaul for their leadership in ensuring the reauthorization of the Creating Hope Act.

We urge the U.S. Senate to also pass 21st Century Cures as the Creating Hope Act is currently set to expire on March 14, 2016, one year after the issuance of the third voucher, as provided in statutory text.

The Creating Hope Act has wildly exceeded our hopes.  On Valentines Day, February 14, 2014, the FDA awarded the first Creating Hope Act voucher to BioMarin for Vimizim to treat Morquio A syndrome.

Then, on March 10, 2015, the FDA awarded the second voucher to United Therapeutics for Unituxin, the most exciting advance in neuroblastoma, a pediatric cancer,  in 40 years, according to the New England Journal of Medicine.

The third voucher was awarded to Asklepion Pharmaceuticals for the approval of Cholbam to treat kids with rare bile acid synthesis disorders.

In addition, two vouchers have now sold at attractive valuations.  BioMarin sold its voucher on July 31, 2014 to Sanofi/Regeneron for $67.5M.  Then, on November 19, 2014, Knights Therapeutics sold to Gillead, a tropical disease voucher, which has the same valuation as a pediatric voucher, for a whopping $125M!

These valuations are proof of the principle that good risk adjusted returns on investment can be made by investing in drug development for pediatric rare diseases, including cancer. These successes are already having a profound impact on the landscape of pediatric cancer research.

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