FDA News

FDA grants breakthrough therapy designation for novel psychotropic agent

Antony Loebel, MD
Antony Loebel

The FDA has granted breakthrough therapy designation for SEP-363856, a novel agent for the treatment of people with schizophrenia, according to a press release.

SEP-363856 is the first treatment for schizophrenia that does not bind to dopamine 2 (D2) or serotonin 2A (5-HT2A) receptors, and is believed to activate trace amine-associated receptor 1 (TAAR1) in addition to serotonin 1A (5-HT1A) receptors, according to the release.

“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,” Antony Loebel, MD, President and CEO at Sunovion, said in the release. “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."

This designation is based on positive data from a phase 2 study and a 6-month, open-label, safety and tolerability extension study, according to the release.

In the phase 2, randomized, placebo-controlled study, SEP-363856 met its primary endpoint, showing that hospitalized patients with acute exacerbation of schizophrenia treated with the drug had statistically significant and clinically meaningful improvement in the Positive and Negative Syndrome Scale (PANSS) total score compared with placebo after 4 weeks of treatment (–17.2 vs. –9.7; P = .001). In addition, patients treated with SEP-363856 also demonstrated improvement in overall illness severity as well as all major PANSS subscales. It was also generally well-tolerated and had similar discontinuation rates to placebo.

Antony Loebel, MD
Antony Loebel

The FDA has granted breakthrough therapy designation for SEP-363856, a novel agent for the treatment of people with schizophrenia, according to a press release.

SEP-363856 is the first treatment for schizophrenia that does not bind to dopamine 2 (D2) or serotonin 2A (5-HT2A) receptors, and is believed to activate trace amine-associated receptor 1 (TAAR1) in addition to serotonin 1A (5-HT1A) receptors, according to the release.

“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,” Antony Loebel, MD, President and CEO at Sunovion, said in the release. “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."

This designation is based on positive data from a phase 2 study and a 6-month, open-label, safety and tolerability extension study, according to the release.

In the phase 2, randomized, placebo-controlled study, SEP-363856 met its primary endpoint, showing that hospitalized patients with acute exacerbation of schizophrenia treated with the drug had statistically significant and clinically meaningful improvement in the Positive and Negative Syndrome Scale (PANSS) total score compared with placebo after 4 weeks of treatment (–17.2 vs. –9.7; P = .001). In addition, patients treated with SEP-363856 also demonstrated improvement in overall illness severity as well as all major PANSS subscales. It was also generally well-tolerated and had similar discontinuation rates to placebo.