After the U.S. Food and Drug Administration (FDA) issued a refuse-to-file letter for Roluperidone, its schizophrenia drug, Massachusetts-based biotech company Minerva Neurosciences revealed it experienced a significant decline in its stock price.
The company had sent the new drug application (NDA) for Roluperidone, for treating schizophrenia-related symptoms in patients suffering from the disorder. However, the FDA wasn’t impressed, even though the agency has stated that Minerva is within its rights to request a Type A meeting for discussion of the content of the refuse-to-file letter.
Among the adverse symptoms of schizophrenia are social withdrawal, a total lack of motivation and flat emotional affect, all of which significantly impact the patient’s quality of life. As such, the need for therapy targeting these negative symptoms is quite pressing.
Before this news was announced, GlobalData had assumed that roluperidone would become available for sale in the U.S. in the third quarter of 2023. Sales predictions were north of $70 million by 2031.
Remy Luthringer, Minerva CEO, stated, “We are disappointed that the FDA has not accepted our NDA for roluperidone. Our goal remains to provide a new and much needed therapeutic option to help patients and their families, since there are currently no approved therapies to treat negative symptoms of schizophrenia in the United States. The company intends to request a Type A meeting and looks forward to continued discussions with the FDA.”
The refuse-to-file letter isn’t the first obstacle Minerva has faced in roluperidone’s development; two FDA Type C meetings regarding the drug have also taken place, with the first one taking place in November 2020 and the second in March this year.
Minerva’s NDA is backed up by results from two clinical trials, one of which took place outside the U.S. However, the big stumbling block was the fact that the drug couldn’t meet the primary efficacy criteria in the U.S.-based study (MIN-101C07). This proved to be a critical point, as this trial was the one to indicate a change in symptoms factor score that was statistically significant.
The FDA didn’t disclose the specific reasons behind the refuse-to-tile letter. However, the agency did highlight two key topics in this regard in the recent Type C meeting: the drug’s effectiveness in treating the symptoms and the extent to which the data collected outside the U.S. is applicable to patients in the country. So it’s entirely possible that the FDA wasn’t convinced by Minerva’s response to these topics in its filing.
Even if Minerva succeeds in addressing the issues raised by the FDA, their drug still has more hurdles to face. Even though key opinion leaders (KOLs) concur that the market for schizophrenia treatments lacks novel therapies, they are still concerned about the small number of patients who can benefit from the roluperidone treatment. Moreover, they believe that they’ll not be too adamant about switching patients from conventional antipsychotic therapy to roluperidone because of the higher risks of relapses from the withdrawal of antipsychotic use.
