FDA Grants Breakthrough Designation to Novel Schizophrenia Drug


The investigative drug SEP-363856 has been evidenced in a phase 2 trial to reduced hospitalized patient symptoms and severity.


The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to novel investigative therapy SEP-363856 for the treatment of schizophrenia.

The Sunovion and PsychoGenics psychotropic has been given an expedited pathway toward regulatory approval based on the findings of a pivotal phase 2 trial presented at the Annual Meeting of the American College of Neuropsychopharmacology (ACNP) in December last year, as well as from a six-month, open-label, safety and tolerability extension trial.

The registration study, SEP 361-201, was a randomized, placebo-controlled, double-blind trial which assessed for a primary endpoint of statistically significant and clinically meaningful improvement in Positive and Negative Syndrome Scale (PANSS) total score versus placebo in treated patients hospitalized with acute schizophrenia exacerbation. At the end of 4 weeks, treated patients reported a significant and meaningful improvement in PANSS versus place (-17.2 vs -9.72, respectively; P= .001).

Patients on the investigative drug also showed improvement in overall illness severity assess by the Clinical Global Impression Scale for severity (P< .001). The therapy was found to be generally well tolerated among treated patients, with similarities to placebo in discontinuation rates, restlessness symptom rates, and changes in metabolic metrics.

The exact mechanism of action for SEP-363856 is unknown, but investigators believe it functions by activating trace amine-associated receptor 1 (TAAR1) and serotonin 1A (5-HT1A) receptors. Currently available antipsychotic therapies bind to either dopamine 2 (D2) or serotonin 2A (5-HT2A) receptors, which is considered to mediate their effect.

The investigative therapy is also being studied for Parkinson disease psychosis and other indications, as part of a global development program. It’s currently evidenced to be considered suitable for once-daily administration.

As a driver of chronic psychiatric abnormalities that affect about 2.4 million people in the US, schizophrenia requires greater, more significant daily therapies. Anthony Loebel, MD, president and chief executive officer at Sunovion, hopes that can be achieved with SEP-363856.

“Breakthrough Therapy Designation underscores the potential of SEP-363856 as a novel treatment for patients with schizophrenia, for whom few major advances in treatment have occurred since the advent of antipsychotic pharmacotherapy in the 1950s,” Loebel said in a statement. “Investigational studies to further evaluate the clinical benefit of SEP-363856 are in progress, and we look forward to working closely with the FDA on this important potential new therapy."

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