Designed as a small molecule vasopressin 1a (V1a) receptor antagonist, balovaptan is reported to have demonstrated potential to improve social interaction and communication for people with ASD.
The FDA has granted the designation based on efficacy data obtained during the Phase II VANILLA trial of balovaptan in adults suffering from ASD.
According to the results, the drug candidate demonstrated favourable safety and tolerability profile.
It is expected that if these results are confirmed in further studies balovaptan can potentially be the first pharmacotherapy to aid core socialisation and communication symptoms of ASD.
Balovaptan is currently being investigated in another Phase II trial in children and adolescents, with plans for additional studies.
Roche chief medical officer and Global Product Development head Sandra Horning 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.
“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.”
The firm has also received positive recommendation from the EU Committee for Medicinal Products for Human Use (CHMP) for the use of its Hemlibra (emicizumab) during routine prophylaxis of bleeding episodes in patients with haemophilia A with factor VIII inhibitors.