GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. said their drug GTX-102 received the orphan drug and rare pediatric disease designations in the U.S. to treat a type of genetic disorder.
GTX-102 is being studied for the Angelman syndrome, a rare neurogenetic disorder that causes developmental delay, balance issues, motor impairment and seizures. While no treatment for the syndrome exists, several symptoms of the disorder have the potential to be treated.
The companies announced a partnership in August to develop GTX-102 which is still in preclinical development. The companies will seek U.S. Food and Drug Administration approval next year to study the medicine in human clinical trials.
The designations will help incentivize the development of the medicine so it can reach the market sooner.
The orphan drug designation will allow for a seven-year market exclusivity of the medicine following FDA approval, waiver or partial payment of application fees and tax credits for clinical testing expenses. With the rare pediatric disease designation, the company may receive a transferrable priority review voucher which can be used to shorten the time the FDA takes to review a medicine's marketing application.
