The FDA has granted Breakthrough Therapy designation to GC4419, a highly selective and potent small molecule dismutase mimetic, for the reduction of the duration, incidence, and severity of severe oral mucositis (SOM) induced by radiation therapy with or without systemic therapy.
Approximately 70 percent of patients with head and neck cancer receiving radiotherapy develop SOM.
Breakthrough Therapy designation for GC4419 was granted based on the data from a 223-patient, double blind, randomized, placebo-controlled, phase IIb clinical trial in patients with head and neck cancer. In the trial, GC4419 reduced the duration of SOM from 19 days to 1.5 days (92%), the incidence of SOM through completion of radiation by 34 percent, and the severity of patients' oral mucositis by 47 percent, while demonstrating acceptable safety when added to a standard radiotherapy regimen.
GC4419 has also received Fast Track designation for the reduction of the severity and incidence of radiation and chemotherapy-induced oral mucositis.
Breakthrough Therapy designation is designed to expedite the development and review of a therapy intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.