The U.S. Food and Drug Administration (FDA) has granted the Swiss drug company Roche a rare ‘breakthrough therapy’ designation for a drug that may ease some features of autism.
The breakthrough designation means that the drug, balovaptan, can move more quickly to FDA approval. Experts are hopeful about the drug’s new status but say the tests used to assess it are less than ideal and the drug’s mode of action is a puzzle.
In Roche’s trial of balovaptan in adults with autism, 223 men on the spectrum took 1.5, 4 or 10 milligrams of the drug or a placebo every day for 12 weeks.
Caregivers rated the men’s social abilities on the Social Responsiveness Scale 2 (SRS) — the trial’s primary outcome measure — before and after the trial. Investigators also administered a scale called the Vineland Adaptive Behavior Scales, which measures how an individual adapts to social changes and navigates life’s day-to-day demands.
The trial showed no benefit for the drug on the SRS, but the men who took the drug scored four or five points higher on the Vineland Adaptive Behavior Scales than did those who got the placebo. The improvement reflects a small but clinically significant effect on social interaction and communication, says Federico Bolognani, the Roche investigator who led the trial. It is unclear why the participants showed improvement on this measure but not on the SRS.
