United Therapeutics nabs priority review voucher, approval for rare pediatric cancer drug
- United Therapeutics' Unituxin (dinutuximab) is the first approved therapy for treatment of high-risk neuroblastoma, a condition that mainly affects children under five.
- Three years after treatment with Unituxin as part of combo treatment, 63% of study participants were not only alive, but free of tumor growth, compared with 46% of patients in the control arm.
- In addition to FDA approval of Unituxin, United Therapeutics also received a priority review voucher worth tens of millions of dollars.
Each year, roughly 650 people are diagnosed with neuroblastoma, mainly young children. Even with aggressive therapy that includes surgery, chemotherapy and radiation, long-term survival rates are only in the 40% to 50% range.
Updated analysis has now shown that Unituxin-treated patients have experienced a 73% survival rate, compared with 58% of those treated with standard therapy. Unituxin's ability to extend survival over a three-year period marks a significant step forward in the treatment of this deadly disease; however, Unituxin is associated with severe pain that often requires treatment with narcotics—and for this reason comes with a black box warning.
Now for part two: The priority review voucher is highly valued throughout the industry and especially useful for companies that are involved in a tight competition to bring a similar therapy to market. Having a priority review voucher can decrease FDA review time by 40% from 10 months to six months, and companies have paid up to $125 million for them.