Dive Brief:
- 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.
Dive Insight:
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.