Right now, there are two medications approved by the FDA for treating symptoms of autism. Both are antipsychotics and neither one targets the core deficits behind autism itself. Drugs that specifically help with the core challenges of autism have been elusive so far.
But one Swiss drug-making company, Roche, may be in the process of changing that: they have produced a medication called Balovaptan, which has been showing some promise in improving social function and communication skills in autistic people.
In fact, the U.S. FDA has granted this drug breakthrough therapy designation. This means that it’s possible the drug will be able to hit the market faster than it would have otherwise.
Balovaptan is what’s known as a vasopressin 1a (V1a) receptor antagonist and, according to Fierce Biotech, works by blocking activity in the V1a vasopressin receptor subtype. Vasopressin is a neuropeptide that facilitates communication between neurons and is involved with social bonding. Increased amounts of it in the blood of autistic people has been linked to better socialization.
If that’s the case, why are researchers trying to block activity? They believe that blocking it at one receptor will improve social abilities, whereas increasing it overall could potentially lead to aggression.
Balovaptan is currently undergoing phase II clinical trials, examining its effects on kids and teens with autism, and it will go through further studies once the current one is done.
“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 Roche’s chief medical officer Sandra Horning. “We look forward to working closely with the FDA in hope that we can bring this medicine to these individuals as quickly as possible.”
According to Reuters, the drug’s expected filing date at the moment is sometime after 2020.
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