This article is more than 5 years old. Information may no longer be current.
FDA grants orphan drug designation to tazemetostat for treatment of malignant rhabdoid tumors
Click Here to Manage Email Alerts
The FDA granted orphan drug status to tazemetostat for the treatment of malignant rhabdoid tumors, according to a press release from the agent’s manufacturer.
The designation applies to use of tazemetostat (Epizyme), an EZH2 inhibitor, for use in patients with INI1-negative malignant rhabdoid tumors, as well as those with SMARCA4-negative malignant rhabdoid tumors of the ovary.
“Malignant rhabdoid tumors are rare and aggressive cancers with poor outcomes. There have not yet been any targeted drugs developed specifically to treat these patients," Peter Ho, MD, PhD, chief medical officer of Epizyme, said in the press release. “In an ongoing phase 1 study, tazemetostat has demonstrated encouraging clinical activity and an acceptable safety profile in patients with these severe types of cancer. We believe tazemetostat has the potential to become an important targeted therapy for patients with malignant rhabdoid tumors and we are working aggressively to execute our clinical development program.”
In December 2015, Epizyme launched a phase 2 study in adults and a phase 1 study in children with genetically-defined solid tumors. These trials included patients with rhabdoid tumors, other INI1-negative tumors and synovial sarcoma.
Interim data from the phase 2 study likely will be presented later this year, according to the press release.
Tazemetostat also is being investigated in a five-arm phase 2 study for the treatment of patients with non-Hodgkin’s lymphoma.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.