A Swiss drugs firm has been granted breakthrough therapy designation for an autism treatment.
The US Food and Drug Administration (FDA) handed the designation to Roche for its potential new autism medication Balovaptan.
Roche’s chief medical officer Sandra Horning: she says the firm wants to make Balovaptan available “as quickly as possible”
The FDA grants the designation to speed up drug development for serious or life-threatening conditions.
Substantial improvements
Before it grants the designation, the FDA also demands that early evidence shows the drug could achieve substantial improvements over existing treatments.
Balovaptan belongs to a class of drugs known as vasopressin receptor antagonists.
These drugs affect the brain. Scientists think that certain receptors of the hormone vasopressin in the brain help to regulate social and aggressive behaviours.
Social skills and communication
Investigators assessing Balovaptan in autism have said the drug shows the potential to improve social skills and communication.
Roche says research evidence suggests vasopressin receptor antagonists can improve the social behaviours that people with autism often find difficult.
The FDA granted the breakthrough designation based on results from a study on adults with autism.
These results showed the drug was safe and that patients tolerated it well.
Sandra Horning is Roche’s chief medical officer and head of global product development.
She said: “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.”
First autism treatment for core symptoms
If the FDA approves the drug it could become the first autism treatment to target the condition’s core symptoms.
Other medications used to treat autism, such as antipsychotics, target secondary symptoms such as aggression.
Trials of Balovaptan were presented at the International Meeting for Autism Research (IMFAR) in May 2017.
A phase II trial investigating Balovaptan in children and adolescents with autism is ongoing.
Drug companies usually perform phase II trials on groups of around 100 to 300. They assess a drug’s effectiveness, as well as continuing to test its safety.
Related:
- Scientists near to predicting aggression
- Concern over chemical restraint
- Social difficulty traced to brain region
Published: 5 February 2018
