The U.S. Food and Drug Administration (FDA) has on January 16th 2019 granted orphan drug status to RTT’s lead product candidate Emcitate® for the treatment of MCT8 deficiency, also known as the Allan Herndon Dudley Syndrome.
“The granting of orphan drug status in the U.S. is yet another important step in our mission to bring Emcitate to market. We believe our product holds great potential to become the first approved pharmaceutical for the treatment of patients with MCT8 deficiency, a very rare and severe condition for which there are currently no available treatment options.”, said Dr. Peder Walberg, the CEO of RTT.
In the U.S., orphan drug status is granted to investigational therapies aimed to treat rare diseases affecting fewer than 200,000 people and gives access to a number of incentives aimed to advance clinical development towards regulatory approval, including fee waivers, free regulatory advice and 7 years market exclusivity following approval. Emcitate already holds a corresponding oprhan drug designation for the treatment of MCT8 deficiency from the European Union.