The Creating Hope Act, a US bill approved in 2012, created the rare pediatric disease priority review voucher (PRV) program, incentivizing drug development for kids with life-threatening illnesses. This program has been recently reauthorized.
Under this program, a sponsor that develops a drug for a rare pediatric disease and receives FDA approval also qualifies for a fully transferable voucher. The PRV grants accelerated FDA review of a drug candidate, for any indication, reducing the review period to 6 months and potentially gaining early market access.
To date, 12 out of 25 PRVs received for pediatric indications have been sold for a cumulative sale price of USD 1.8 billion.
Last September Oncoheroes announced that the FDA granted the rare pediatric disease designation (RPDD) to volasertib, an investigational treatment for rhabdomyosarcoma. The RPDD qualifies Oncoheroes to receive fast track review, and a priority review voucher (PRV) at the time of marketing approval of volasertib.
Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. The compound was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia until the company decided to discontinue the compound for strategic reasons. Meanwhile, independent academic groups generated strong data in support of further development of volasertib for rhabdomyosarcoma and, possibly, a few other pediatric cancer indications. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.
