FDA Grants ‘Breakthrough Therapy’ Status To Potential Autism Drug
A major pharmaceutical company says it has received a designation from the Food and Drug Administration to help expedite what could be the first drug to treat the core symptoms of autism.
Swiss drug maker Roche said this week that the FDA has granted its breakthrough therapy designation for the development of balovaptan, a medication with the potential to improve “core social interaction and communication” in those with autism.
The FDA uses the breakthrough therapy designation to speed the development of promising medications for serious conditions. It offers drugmakers added assistance and priority from the federal agency as they work to get a new treatment to market.
Advertisement - Continue Reading Below
There are only two drugs — risperidone and aripripazole — with FDA approval to treat irritability related to autism. No drugs are currently approved to address core symptoms of the condition.
Results from a clinical trial in adults with autism released last year indicate that balovaptan was successful in helping mediate challenging social behaviors and was safe and well tolerated. Another trial looking at children and adolescents on the spectrum is underway and additional studies are in the works, Roche said.
“We are very pleased that the FDA has granted breakthrough therapy designation for balovaptan in recognition of its early promise for individuals with ASD,” said Sandra Horning, chief medical officer and head of global product development at Roche. “We look forward to working closely with the FDA in the hope that we can bring this medicine to these individuals as quickly as possible.”