Source: Tarix Orphan LLC & reported by http://www.prnewswire.com/
CAMBRIDGE, Mass., Jan. 18, 2017 /PRNewswire/ -- Tarix Orphan LLC today announced that the U.S. Food and Drug Administration has granted a Rare Pediatric Disease (RPD) designation for the company's drug candidate TXA127 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), a rare genetic skin disorder. There are currently no approved therapies for RDEB and treatment is limited to supportive care.
"The RPD designation for TXA127 in RDEB supplements the Orphan Drug Designation previously granted by the FDA in this indication," said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. "RDEB is a devastating disease. We have been very encouraged by the preliminary animal data we have generated and are looking forward to confirming these results in an additional model through the support of DEBRA International, the European patient organization focused on epidermolysis bullosa."
The FDA defines 'Rare Pediatric Disease' as a disease that primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 persons in the United States. Under the Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval for a drug or biologic for a 'rare pediatric disease' may qualify for a voucher which can be redeemed to receive a priority review of a subsequent marketing application for a different product. The Priority Review Voucher is requested at the time of the marketing application and awarded upon approval of the product. The voucher may only be used once, but may be sold or transferred an unlimited number of times.
Read more: http://www.prnewswire.com/news-releases/tarix-orphan-receives-rare-pediatric-disease-designation-for-txa127-for-recessive-dystrophic-epidermolysis-bullosa-rdeb-300392579.html