Article Source: BioCentury Extra
Published on Tuesday, March 10, 2015
FDA vouchers for United Therapeutics’ Unituxin
FDA approved Unituxin dinutuximab (Ch14.18) from United Therapeutics Corp. (NASDAQ:UTHR) to treat highrisk pediatric neuroblastoma and granted the company a Priority Review voucher through the agency’s Rare Pediatric Disease Priority Review Voucher Program.
Unituxin was approved under Priority Review, and has Orphan Drug status in the U.S. to treat the rare pediatric cancer. United spokesperson Andy Fisher said the company plans to launch Unituxin in the U.S. this summer, and will disclose pricing at that time. The drug is under EMA review in the EU, where it also has Orphan designation.
Fisher said United is evaluating options for how to use the transferable voucher.
United is the second company to receive a voucher since the pediatric program was created in 2012. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) received a voucher for Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPSIVA, Morquio’s syndrome), which it sold to Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) for $67.5 million (see BioCentury, Aug. 4, 2014).
Unituxin, a chimeric mAb targeting GD2, is indicated in combination with granulocytemacrophage colonystimulating factor (GMCSF), interleukin2 (IL2) and 13cisretinoic acid in patients who have achieved at least a partial response to surgery, chemotherapy and radiation therapy.
Unituxin’s label carries a boxed warning for severe pain, risk of nerve damage and lifethreatening infusion reactions. FDA said United Therapeutics has agreed to postmarketing requirements and commitments, but the company declined to disclose them.
FDA based its approval on results from the 226patient Phase III ANBL0032 trial, conducted by the Children’s Oncology Group, in which Unituxin improved eventfree survival (EFS) and overall survival (OS) (see BioCentury Extra, May 28, 2009).
In a statement, Kids v Cancer noted Unituxin is only the third drug in more than 20 years to receive initial FDA approval to treat a pediatric cancer.
Last year, Nancy Goodman, founder and executive director of the not-for-profit, said she expected interest in the pediatric voucher incentive to accelerate after FDA released a draft guidance on the program, which the agency issued in November.
United shares gained $3.40 to $163.84 on Tuesday.
PDF Format of Article: BioCentury Extra: FDA vouches for United Therapeutics’ Unituxin, March 10, 2015