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Marinus genetic epilepsy drug meets main goal of late-stage study

September 14, 2020

Marinus Pharmaceuticals Inc said on Monday its experimental treatment for a rare type of genetic epilepsy called CDKL5 deficiency disorder (CDD) met the main goal of reducing seizure frequency in patients in a late-stage trial.

CDD is characterized by difficult-to-control seizures and severe neuro-developmental impairment.

Patients who received the company’s oral drug, ganaxolone, showed a 32.2% reduction in seizure frequency, compared with a 4% decrease in those who received a placebo, meeting the study’s main goal.

The drug, however, did not meet statistical significance across the secondary goals of the trial, which had enrolled 101 patients.

There are currently no approved therapies for CDD, and most children affected by it cannot walk, talk, or feed themselves, Marinus said.

In addition to CDD, ganaxolone is also being tested for refractory status epilepticus, or RSE, a condition in which people fail to respond to two or more commonly used antiepileptic drugs.

Earlier on Monday, the company announced a five-year cost-sharing contract with the Biomedical Advanced Research and Development Authority (BARDA) to support a late-stage study for the treatment of RSE.

Marinus said it plans on filing a marketing application for ganaxolone for the treatment of CDD with the U.S. health regulator in mid-2021.

The Radnor, Pennsylvania-based company said it would also file for EU approval for the drug by the end of the third quarter of 2021.

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