United Therapeutics wins FDA OK for rare kids' cancer drug, plus a priority review voucher

The FDA has approved United Therapeutics' ($UTHR) Unituxin (dinutuximab) to treat pediatric patients with high-risk cases of neuroblastoma. And the regulatory win comes with a priority review vouch that may well be worth tens of millions of dollars.

There are only about 650 new cases of neuroblastoma in the U.S. every year, with children under 5 as primary victims of a disease that currently comes with a 40% to 50% chance of longterm survival. The drug is an antibody that binds to the surface of neuroblastoma cells. And the agency notes that the data supports its ability to extend survival.

Three years after treatment, says the agency, 63% of the trial subjects getting a combo treatment including Unituxin were alilve and free of tumor growth or recurrence, compared to only 46% in the control arm. The most recent update on the data brought the rate of survival up to 73% in the combo group.

The regulatory win comes with a bonus from the FDA for developing a drug for a rare pediatric condition. United gets a priority review voucher that can be sold to anyone looking for a regulatory shortcut. Sanofi ($SNY) and Regeneron ($REGN) bought one for their PCSK9 program (a $67.5 million deal with Biomarin) that allowed them to leapfrog Amgen in the race to the FDA. And Gilead ($GILD) paid $125 million for their voucher, which has yet to be assigned to a drug program. A priority review voucher can be used to cut the FDA's process for new drugs down from the standard 10 months to 6 months, a valuable shortcut in the biopharma world.

Dr. Richard Pazdur

"Unituxin marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "Unituxin fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma."

- here's the release on the approval
- here's the release on the voucher